{"id":18528,"date":"2022-08-19T16:58:05","date_gmt":"2022-08-19T11:28:05","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=18528"},"modified":"2023-02-10T14:41:06","modified_gmt":"2023-02-10T09:11:06","slug":"aav-vectors-in-gene-therapy-landscape","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape","title":{"rendered":"AAV Vectors in Gene Therapy: How Recent Clinical Advances are Unraveling New Potentials?"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_76 counter-hierarchy ez-toc-counter ez-toc-white ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-69fc5f81c0c3e\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-69fc5f81c0c3e\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Adeno-Associated_Virus_AAV_Vector_Designs\" >Adeno-Associated Virus (AAV) Vector Designs<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#AAV_as_a_Gene_Therapy_Vector\" >AAV as a Gene Therapy Vector<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Promising_AAV_Vectors_for_Gene_Therapy_in_Pipeline\" >Promising AAV Vectors for Gene Therapy in Pipeline<\/a><ul class='ez-toc-list-level-3' ><li class='ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#GS010_Gensight_Biologics\" >GS010: Gensight Biologics<\/a><ul class='ez-toc-list-level-4' ><li class='ez-toc-heading-level-4'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Product_Development_Activities\" >Product Development Activities<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Valoctocogene_Roxaparvovec_BioMarin_Pharmaceutical\" >Valoctocogene Roxaparvovec: BioMarin Pharmaceutical<\/a><ul class='ez-toc-list-level-4' ><li class='ez-toc-heading-level-4'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Product_Development_Activities-2\" >Product Development Activities<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#NFS-01_Neurophth_Biological_Tech\" >NFS-01: Neurophth Biological Tech<\/a><ul class='ez-toc-list-level-4' ><li class='ez-toc-heading-level-4'><a class=\"ez-toc-link ez-toc-heading-9\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Product_Development_Activities-3\" >Product Development Activities<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-10\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#AAV-CNGA3_MeiraGTx\" >AAV-CNGA3: MeiraGTx<\/a><ul class='ez-toc-list-level-4' ><li class='ez-toc-heading-level-4'><a class=\"ez-toc-link ez-toc-heading-11\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#Product_Development_Activities-4\" >Product Development Activities<\/a><\/li><\/ul><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-12\" href=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\/#What_Lies_Ahead\" >What Lies Ahead?<\/a><\/li><\/ul><\/nav><\/div>\n\n<p>There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical-stage experimental therapy techniques. The capacity to create recombinant AAV particles devoid of viral genes but carrying DNA sequences of interest for diverse therapeutic purposes has thus far proven to be one of the safest gene therapy techniques.<\/p>\n\n\n\n<p><a href=\"https:\/\/www.delveinsight.com\/report-store\/adeno-associated-virus-vectors-in-gene-therapy-market\">Adeno-associated virus (AAV)<\/a>, discovered 5 decades back in&nbsp; mid-1960s from laboratory adenovirus (AdV) preparations was a result of serendipity. A few research organizations set out to grasp fundamental AAV biology out of sheer scientific interest and without recognizing its enormous potential as a human gene therapy platform. Several essential characteristics of AAV were described over the first 15-20 years of AAV study, including its genomic configuration and composition, DNA replication and transcription, virulence, and virion assembly.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-adeno-associated-virus-aav-vector-designs\"><span class=\"ez-toc-section\" id=\"Adeno-Associated_Virus_AAV_Vector_Designs\"><\/span><strong>Adeno-Associated Virus (AAV) Vector Designs<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>The package size of the expression cassette that will be inserted between the two inverted terminal repeats (ITRs) is the most important factor to consider in the logical rAAV vector design. As a starting point, it is widely considered that anything less than 5 kb (including viral ITRs) is enough. Attempts to generate rAAV vectors with packaging cassettes larger than 5 kb result in a significant drop in viral production yields or transgene recombination (truncations). As a result, big coding sequences, such as full-length dystrophin genes, will not be packed well in AAV vectors.<\/p>\n\n\n\n<p><strong>Adeno-associated virus type-2<\/strong><strong> (<\/strong>AAV2) was one of the first AAV serotypes to be found and described, including its genomic sequence. Because of this early work&#8217;s deep understanding of AAV2 biology, most <a href=\"https:\/\/www.delveinsight.com\/report-store\/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight\">rAAV vectors<\/a> developed today use the AAV2 ITRs in their AAV vector designs. A mammalian promoter, gene of interest, and a terminator are normally inserted between the ITRs.&nbsp;<\/p>\n\n\n\n<p>In many circumstances, robust, constitutively active promoters are desired for high-level gene expression. The <strong>cytomegalovirus (<\/strong><strong>C<\/strong><strong>MV) promoter\/enhancer, elongation factor 1a (EF1a), simian virus 40 (SV40), chicken -actin, and CAG (CMV, chicken -actin, rabbit -globin)<\/strong> are all examples of this type of promoter. In most cell types, all of these promoters enable constitutively active, high-level gene expression. Because some of these promoters are susceptible to silencing in specific cell types, this factor must be considered for each application.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"AAV_as_a_Gene_Therapy_Vector\"><\/span><strong>AAV as a Gene Therapy Vector<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Any viral vector must take various factors into account, including the capacity to adhere to and penetrate the target cell, effective transfer to the nucleus, sustained expression in the nucleus, and a general lack of toxicity. All of these conditions have been met with great success by AAV vectors. Furthermore, a number of improvements have contributed to improve their usability.&nbsp;<\/p>\n\n\n\n<p>Several factors have influenced the creation of modern <a href=\"https:\/\/www.delveinsight.com\/report-store\/adeno-associated-virus-vectors-in-gene-therapy-market\">AAV vector gene therapy<\/a>, most notably the wild-type virus&#8217;s lack of pathogenicity and persistence. Because of the limited size of the AAV genome and concerns about Rep&#8217;s possible impact on cellular gene expression, AAV vectors that do not encode Rep and lack the cis-active IEE required for frequent site-specific integration were developed. The ITRs are retained since they are necessary for packing. As a result, modern recombinant AAV (rAAV) vector gene therapy are mostly found as extrachromosomal elements.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Promising_AAV_Vectors_for_Gene_Therapy_in_Pipeline\"><\/span><strong>Promising AAV Vectors for Gene Therapy in Pipeline<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<figure class=\"wp-block-image size-full is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160354\/AAV-vectors-in-gene-therapy-Assessment-by-Product-Type.png\" alt=\"AAV Vectors in gene therapy by product type\" class=\"wp-image-18549\" width=\"681\" height=\"477\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160354\/AAV-vectors-in-gene-therapy-Assessment-by-Product-Type.png 681w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160354\/AAV-vectors-in-gene-therapy-Assessment-by-Product-Type-300x210.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160354\/AAV-vectors-in-gene-therapy-Assessment-by-Product-Type-150x105.png 150w\" sizes=\"(max-width: 681px) 100vw, 681px\" \/><figcaption class=\"wp-element-caption\"><strong>AAV Vectors in Gene Therapy Therapeutics Assessment: By Product Type<\/strong><\/figcaption><\/figure>\n\n\n\n<p>AAV has been found to be a very stable vector, able to survive extensive temperature and pH variations with little to no activity loss. To date, the sole constraint appears to be the concentration with which it is currently being formulated and optimized at about 5 x 1013 particles per milliliter. With the return of clinical usage, this formulation restriction will most certainly be surpassed in the near future. However, the strong stability of these vectors affords enough possibilities to test various administration routes and specific delivery techniques.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-1024x478.png\" alt=\"AAV Vectors in Gene Therapy By RoA\" class=\"wp-image-18550\" width=\"1024\" height=\"478\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-1024x478.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-300x140.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-150x70.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-768x359.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-1536x717.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart-1568x732.png 1568w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160455\/AAV-vectors-in-gene-therapy-Assessment-by-Route-of-Administration-pie-chart.png 1679w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\"> <strong>AAV Vectors in Gene Therapy Therapeutics Assessment: By RoA<\/strong> <\/figcaption><\/figure>\n\n\n\n<p>Currently, more than <strong>70 companies<\/strong> are evaluating <strong>235+<\/strong> AAV vectors in gene therapy landscape. These <a href=\"https:\/\/www.delveinsight.com\/report-store\/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight\">AAV vectors in gene therapy<\/a> are presently being studied at different stages of development by leading goliaths such as <strong>BioMarin Pharmaceutical, Gensight Biologics, PTC Therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, UniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark Therapeutics (Roche), Sarepta Therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology Medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline Therapeutics, Astellas Pharma, Aspa Therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus Therapeutics, 4D Molecular Therapeutics, <\/strong>and others. <\/p>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-1024x603.png\" alt=\"AAV Vectors in gene therapy Assessment by Stage and Route of Administration\" class=\"wp-image-18547\" width=\"1024\" height=\"603\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-1024x603.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-300x177.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-150x88.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-768x452.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-1536x904.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration-1568x923.png 1568w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19154127\/AAV-Vectors-in-gene-therapy-Assessment-by-Stage-and-Route-of-Administration.png 1877w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\"> <strong>AAV Vectors in Gene Therapy Segmentation:<\/strong> <strong>By Stage and RoA<\/strong><\/figcaption><\/figure>\n\n\n\n<p>Every AAV vector gene therapy candidate has a novel MoA and product type. The most promising AAV vector for gene therapy in the pipeline includes:<\/p>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-1024x428.png\" alt=\"AAV Vectors in Gene Therapy By Product Type and Stage\" class=\"wp-image-18551\" width=\"1024\" height=\"428\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-1024x428.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-300x125.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-150x63.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-768x321.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-1536x642.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type-1568x655.png 1568w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19160649\/AAV-vectors-in-gene-therapy-Assessment-by-Stage-and-Product-type.png 1877w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\">  <strong>AAV Vectors in Gene Therapy Segmentation:<\/strong> <strong>By Product Type and Stage<\/strong> <\/figcaption><\/figure>\n\n\n\n<h3 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"GS010_Gensight_Biologics\"><\/span><strong>GS010: Gensight Biologics<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>LUMEVOQ\u00ae (GS010; Lenadogene Nolparvovec)<\/strong> targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform developed at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address mitochondrial defects using an AAV vector. The gene of interest is introduced into the cell and expressed, resulting in the production of a functional protein which is subsequently shuttled to the mitochondria via particular nucleotidic sequences to restore the missing or defective mitochondrial function. The European Medicines Agency (EMA) approved &#8220;LUMEVOQ&#8221; as the invented name for GS010 (lenadogene nolparvovec) in October 2018.<\/p>\n\n\n\n<p>The European Marketing Authorisation Application for LUMEVOQ\u00ae is currently being reviewed, with the CHMP&#8217;s decision expected in <strong>Q3 2023 <\/strong>as a consequence of an extension granted by the EMA for GenSight&#8217;s replies to its Day 120 queries. Commercialization will begin after approval by the end of <strong>2023<\/strong>.<\/p>\n\n\n\n<h4 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Product_Development_Activities\"><\/span><strong>Product Development Activities<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h4>\n\n\n\n<p><strong>Presentation of GS010 Clinical Data at EUNOS 2022<\/strong><\/p>\n\n\n\n<p>GenSight Biologics stated in June 2022 that it will attend the <strong>EUNOS 2022 meeting<\/strong>, which will be held on June 20-23 in Birmingham, United Kingdom.<\/p>\n\n\n\n<p><strong>European Regulatory Review<\/strong><\/p>\n\n\n\n<p>In April 2022, GenSight Biologics announced that the European Medicines Agency&#8217;s (EMA) Committee for Advanced Therapies (CAT) had granted the company a 6-month extension for responding to Day 120 questions in the regulatory review of <strong>LUMEVOQ\u00ae<\/strong>, GenSight&#8217;s gene therapy for the <a href=\"https:\/\/www.delveinsight.com\/report-store\/lebers-hereditary-optic-neuropathy-lhon-market\">treatment of Leber Hereditary Optic Neuropathy (LHON)<\/a>.<\/p>\n\n\n\n<div>\n<table style=\"border:none; border-collapse:collapse\" width=\"588\">\n\t<colgroup>\n\t\t<col width=\"232\">\n\t\t<col width=\"356\">\n\t<\/colgroup>\n\t<tbody>\n\t\t<tr>\n\t\t\t<td colspan=\"2\" style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#cc223d; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#ffffff\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">General Description: GS 010<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">GS 010<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Generic Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Alias Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Lenadogene nolparvovec; LUMEVOQ; ND4 gene therapy; rAAV2\/2-ND4; RAAV2\/2-ND4 vector; rAAV2\/2<\/span><\/span><\/span><\/span><\/span><\/span><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:'Times New Roman'\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">\u2011<\/span><\/span><\/span><\/span><\/span><\/span><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">ND4 gene therapy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">GenSight Biologics<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Product Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mono<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Therapy Area<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Genetic disorder; Ophthalmology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Primary Indication<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Leber Hereditary Optic atrophy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Highest Stage of Development<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Preregistration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Orphan Drug Designation; Promising Innovative Medicine (PIM) designation (UK MHRA)<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensed From Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensing Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Development Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Institutional Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Route of Administration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Intravitreal<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Target<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mechanism of Action<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene transference; NADH dehydrogenase subunit 4 replacement<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Technology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Molecule Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene therapy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Origin<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Natural Source<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t<\/tbody>\n<\/table>\n<\/div>\n\n\n\n<div style=\"height:30px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p><strong>LUMEVOQ\u00ae Manufacturing Timeline<\/strong><\/p>\n\n\n\n<p><strong>GenSight Biologics<\/strong> reported in April 2022 that the completion of the validation (PPQ) batches for <strong>LUMEVOQ\u00ae<\/strong>, the Company&#8217;s <a href=\"https:\/\/www.delveinsight.com\/report-store\/leber-hereditary-optic-neuropathy-pipeline-insight\">gene therapy for Laber Hereditary Optic Neuropathy treatment<\/a>. The delay is required to undertake operational changes that will avoid the recurrence of problems with the most recent PPQ campaign. The campaign is expected to resume in the fourth quarter of 2022.<\/p>\n\n\n\n<p><strong>Designation<\/strong><\/p>\n\n\n\n<p>GenSight Biologics announced in September 2021 that its gene therapy <strong>LUMEVOQ\u00ae<\/strong> had been designated as a <strong>Promising Innovative Medicine (PIM)<\/strong> by the UK&#8217;s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of vision loss caused by Leber Hereditary Optic Neuropathy (LHON) caused by a confirmed G11778A mutation in the ND4 mitochondrial gene.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Valoctocogene_Roxaparvovec_BioMarin_Pharmaceutical\"><\/span><strong>Valoctocogene Roxaparvovec: BioMarin Pharmaceutical<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>Valoctocogene roxaparvovec <\/strong>is an investigational AAV5 gene therapy under regulatory consideration for the treatment of severe hemophilia A. It is presently in the preregistration stage of development. Hemophilia A is a bleeding condition caused by mutations in the F8 gene, which codes for the clotting protein Factor VIII. Patients with hemophilia A lack&nbsp; Factor VIII in their genetic makeup. For severe instances, frequent intravenous infusions of recombinant or plasma concentrated Factor VIII is usually used.<\/p>\n\n\n\n<p><strong>Valoctocogene roxaparvovec<\/strong> is a gene therapy that combines an adeno-associated virus 5 (AAV5) that codes for human Factor VIII with a human liver-specific promoter that stimulates translation in liver endothelial and sinusoidal cells, where Factor VIII is normally synthesized. The patient would be able to create adequate levels of physiologically active F8 gene by transfecting its functional form into liver cells.<\/p>\n\n\n\n<h4 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Product_Development_Activities-2\"><\/span><strong>Product Development Activities<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h4>\n\n\n\n<p><strong>Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec<\/strong><\/p>\n\n\n\n<p><strong>BioMarin Pharmaceutical Inc. <\/strong>announced in June 2022 that the Committee for Medicinal Products for Human Use (CHMP) had adopted a positive opinion recommending Conditional Marketing Authorization (CMA) for valoctocogene roxaparvovec, an investigational <a href=\"https:\/\/www.delveinsight.com\/report-store\/hemophilia-a2030-market\">gene therapy for adults with severe hemophilia A<\/a>. The European Commission is likely to make a final clearance decision in <strong>Q3 2022<\/strong>, which will be consistent with the CHMP proposal.<\/p>\n\n\n\n<p><strong>Designation<\/strong><\/p>\n\n\n\n<p>In March 2021, the FDA designated valoctocogene roxaparvovec as a <strong>Regenerative Medicine Advanced Therapy (RMAT)<\/strong>. RMAT is a streamlined program designed to speed the development and approval of regenerative medicine therapies, such as the valoctocogene roxaparvovec, that are intended to meet an unmet medical need in individuals suffering from severe diseases. The RMAT designation supplements the Company&#8217;s <strong>Breakthrough Therapy Designation<\/strong>, which it acquired in 2017.<\/p>\n\n\n\n<div>\n<table style=\"border:none; border-collapse:collapse\" width=\"588\">\n\t<colgroup>\n\t\t<col width=\"232\">\n\t\t<col width=\"356\">\n\t<\/colgroup>\n\t<tbody>\n\t\t<tr>\n\t\t\t<td colspan=\"2\" style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#cc223d; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#ffffff\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">General Description: Valoctocogene roxaparvovec<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Valoctocogene roxaparvovec<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Generic Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Alias Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">AAV5-hFVIII-SQ; BMN-270; Factor-VIII-gene-therapy-BioMarin; Roctavian<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">BioMarin Pharmaceutical<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Product Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mono<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Therapy Area<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Genetic disorder; Hematology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Primary Indication<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Haemophilia A<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Highest Stage of Development<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Preregistration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Regenerative Medicine Advanced Therapy, Breakthrough Therapy Designation, Orphan Drug Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensed From Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensing Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Development Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Institutional Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Route of Administration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Intravenous<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Target<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mechanism of Action<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Factor VIII replacement; Gene transference<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Technology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Molecule Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene therapy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Origin<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Natural Source<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t<\/tbody>\n<\/table>\n<\/div>\n\n\n\n<div style=\"height:30px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>In addition to RMAT and Breakthrough Therapy Designation, <strong>BioMarin&#8217;s valoctocogene roxaparvovec<\/strong> has received <strong>Orphan Drug Designation<\/strong> by the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program aims to accelerate the assessment and development of medications that show promise for diagnosing and treating<a href=\"https:\/\/www.delveinsight.com\/blog\/rare-disease-market-drivers\"> rare diseases<\/a> or conditions.<\/p>\n\n\n\n<p><strong>Resubmission of a Biologics License Application to the U.S. Food and Drug Administration (FDA)<\/strong><\/p>\n\n\n\n<p>BioMarin announced in <strong>June 2022<\/strong> that its intended timeframe for resubmitting a Biologics License Application to the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec, commonly known as roctavian, had been moved back to the end of September 2022 &#8211; the original target had been June.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"NFS-01_Neurophth_Biological_Tech\"><\/span><strong>NFS-01: Neurophth Biological Tech<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>NFS-01<\/strong>, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is being developed as an ophthalmic injection for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations.<\/p>\n\n\n\n<h4 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Product_Development_Activities-3\"><\/span><strong>Product Development Activities<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h4>\n\n\n\n<p>The U.S. FDA granted <strong>NFS-01 (NR082, rAAV-ND4)<\/strong> an <strong>Orphan Drug Designation<\/strong> in September 2020, laying the groundwork for speeding the worldwide development of this therapy.<\/p>\n\n\n\n<p>In April 2021, Neurophth reported that the Center for Drug Evaluation (CDE) of China&#8217;s National Medical Products Administration (NMPA) had accepted the Company&#8217;s<strong> Investigational New Drug (IND)<\/strong> application for <strong>NR082<\/strong> for <a href=\"https:\/\/www.delveinsight.com\/report-store\/lebers-hereditary-optic-neuropathy-lhon-epidemiology-forecast\">LHON patients<\/a> with ND4 mutations. It is China&#8217;s first AAV2 gene therapy IND approval.<\/p>\n\n\n\n<div>\n<table style=\"border:none; border-collapse:collapse\" width=\"588\">\n\t<colgroup>\n\t\t<col width=\"232\">\n\t\t<col width=\"356\">\n\t<\/colgroup>\n\t<tbody>\n\t\t<tr>\n\t\t\t<td colspan=\"2\" style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#cc223d; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#ffffff\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">General Description: NFS-01<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NFS-01<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Generic Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Alias Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">AAV2-ND4; AAV2-ND4 Gene Therapy; NFS-01; NR-082; rAAV2 ND4<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Neurophth biological tech<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Product Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mono<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Therapy Area<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Ophthalmology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Primary Indication<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Leber Hereditary Optic atrophy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Highest Stage of Development<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Phase II\/III<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Orphan Drug Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensed From Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensing Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Development Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Institutional Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Huazhong University of Science and Technology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Route of Administration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Intravitreal<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Target<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mechanism of Action<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene transference<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Technology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Molecule Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene therapy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Origin<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Natural Source<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t<\/tbody>\n<\/table>\n<\/div>\n\n\n\n<div style=\"height:30px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>Moreover,<strong> Neurophth<\/strong> secured more than <strong>USD 60 million<\/strong> in round C investment in November 2021 to explore gene treatments for ophthalmic diseases. <strong>CMG-SDIC Capital, Sequoia Capital China, Sunshine Insurance, and China Merchant Bank International Capital<\/strong> are among the company&#8217;s major investors.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"AAV-CNGA3_MeiraGTx\"><\/span><strong>AAV-CNGA3: MeiraGTx<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>AAV-CNGA3<\/strong>, a gene therapy treatment meant to restore cone function, is delivered to the cone receptors at the back of the eye through subretinal injection. It was designed with a synthetic promoter associated with high gene expression to account for the higher quantity of protein required to restore cone function in achromatopsia (ACHM) patients with a CNGA3 gene mutation. MeiraGTx is conducting a Phase I\/II clinical study of AAV-CNGA3 in children aged 3 to 15 years old with ACHM caused by CNGA3 gene mutations.<\/p>\n\n\n\n<h4 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Product_Development_Activities-4\"><\/span><strong>Product Development Activities<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h4>\n\n\n\n<p><strong>MeiraGTx<\/strong> announced in January 2021 that the US Food and Drug Administration (FDA) had given Fast Track designation to their AAV-CNGA3 gene therapy product candidate for the <a href=\"https:\/\/www.delveinsight.com\/report-store\/achromatopsia-market\">treatment of achromatopsia (ACHM)<\/a> caused by CNGA3 gene mutations.<\/p>\n\n\n\n<p><strong>MeiraGTx<\/strong> and <strong>Janssen Pharmaceuticals<\/strong> began exploring AAV-CNGA3 in January 2019 as part of larger cooperation to develop and market gene therapies for the treatment of hereditary retinal disorders.<\/p>\n\n\n\n<div>\n<table style=\"border:none; border-collapse:collapse\" width=\"588\">\n\t<colgroup>\n\t\t<col width=\"232\">\n\t\t<col width=\"356\">\n\t<\/colgroup>\n\t<tbody>\n\t\t<tr>\n\t\t\t<td colspan=\"2\" style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#cc223d; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#ffffff\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">General Description: AAV- CNGA3<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">AAV- CNGA3<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Generic Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Alias Name<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">A-003 &#8211; MeiraGTx; AAV-CNGA3; AAV2\/8-hG1.7p.coCNGA3; CNGA3-gene-therapy-AGTC; Recombinant-adeno-associated-virus-vector-expressing-GNGA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">MeiraGTx<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Product Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mono<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Therapy Area<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Ophthalmology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Primary Indication<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Achromatopsia<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Highest Stage of Development<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Phase I\/II<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Orphan Drug designation by the FDA and EMA, Rare Pediatric Disease designation, Fast Track designation<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensed From Company<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Licensing Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Development Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Janssen Research &amp; Development<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Institutional Partner<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Route of Administration<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Subretinal<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Target<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Mechanism of Action<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene transference<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Technology<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">NA<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Molecule Type<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Gene therapy<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t\t<tr>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#f2f2f2; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:232px\">\n\t\t\t<p style=\"line-height:1.3800000000000001\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:700\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Drug Origin<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t\t<td style=\"border-bottom:1px solid #808080; vertical-align:top; background-color:#ffffff; padding:7px 7px 7px 7px; border-top:1px solid #808080; border-right:1px solid #808080; border-left:1px solid #808080; width:356px\">\n\t\t\t<p style=\"line-height:1.3800000000000001; text-align:justify\"><span style=\"overflow:hidden\"><span style=\"overflow-wrap:break-word\"><span style=\"font-size:10pt; font-variant:normal; white-space:pre-wrap\"><span style=\"font-family:Arial\"><span style=\"color:#000000\"><span style=\"font-weight:400\"><span style=\"font-style:normal\"><span style=\"text-decoration:none\">Natural Source<\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/span><\/p>\n\t\t\t<\/td>\n\t\t<\/tr>\n\t<\/tbody>\n<\/table>\n<\/div>\n\n\n\n<div style=\"height:30px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p><strong>MeiraGTx <\/strong>announced in August 2018 that the U.S. FDA&#8217;s Offices of Orphan Products Development and Pediatric Therapeutics had granted <strong>rare pediatric disease designation<\/strong> to the Company&#8217;s gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to CNGA3 gene mutations.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"What_Lies_Ahead\"><\/span><strong>What Lies Ahead?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Researchers have used gene and other nucleic acid transfer into cells for more than four decades as a research tool. The hunt for real <a href=\"https:\/\/www.delveinsight.com\/whitepaper-newsletter\/gene-therapy-outlook\">gene treatments<\/a> has been pushed by our increased understanding of the genetic components underlying particular diseases. New areas of study have gradually uncovered other possible avenues for gene delivery to be used therapeutically. Furthermore, the limits of current small molecule and protein therapy platforms have fueled the hunt for new therapeutic platforms that address such shortcomings. Gene treatments address all of these obstacles, particularly target accessibility. As a result, the hunt for safe and effective gene delivery methods has been a key emphasis in pharmaceutical research and development, and will ideally mark a paradigm change in how we approach disease-state intervention.Adeno associated virus was developed over 50 years ago and has since evolved into one of the most important gene delivery vectors in clinical research. AAV may become the vector of choice for most gene therapy applications due to its unique biology, simple structure, and lack of known disease correlations. <a href=\"https:\/\/www.delveinsight.com\/whitepaper-newsletter\/aav-vectors-in-gene-therapy-newsletter\">Gene therapy using rAAV<\/a> has been shown to be safe and well-tolerated in almost every clinical context where it has been employed. These studies, along with basic biology research, have revealed numerous aspects of this vector that might be used in future initiatives.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><a href=\"https:\/\/www.delveinsight.com\/whitepaper-newsletter\/aav-vectors-in-gene-therapy-newsletter\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-1024x256.png\" alt=\"AAV Vectors in Gene Therapy Market Scenario\" class=\"wp-image-18531\" width=\"1024\" height=\"256\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-1024x256.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-300x75.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-150x38.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-768x192.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-1536x384.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario-1568x392.png 1568w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142156\/AAV-Vectors-in-gene-therapy-market-scenario.png 1584w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><\/a><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical-stage experimental therapy techniques. The capacity to [&hellip;]<\/p>\n","protected":false},"author":14,"featured_media":18530,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[16988,19656,19657,19658,258,284,5748],"industry":[17225],"therapeutic_areas":[17245,17228,17244,17234],"class_list":["post-18528","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-articles","tag-aav-gene-therapies","tag-aav-vectors","tag-aav-vectors-in-gene-therapy","tag-achromatopsia","tag-gene-therapy","tag-hemophilia-a","tag-leber-hereditary-optic-neuropathy-treatment","industry-pharmaceutical","therapeutic_areas-neurology","therapeutic_areas-oncology","therapeutic_areas-ophthalmology","therapeutic_areas-rare-diseases"],"acf":[],"yoast_head":"<!-- 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improve the therapeutics segment.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.delveinsight.com\/blog\/aav-vectors-in-gene-therapy-landscape\" \/>\n<meta property=\"og:site_name\" content=\"DelveInsight Business Research\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/DelveInsight-1423323754607782\/\" \/>\n<meta property=\"article:published_time\" content=\"2022-08-19T11:28:05+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2023-02-10T09:11:06+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2022\/08\/19142127\/aav-vectors-in-gene-therapy-landscape.png\" \/>\n\t<meta property=\"og:image:width\" content=\"772\" \/>\n\t<meta property=\"og:image:height\" content=\"482\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"Sandeep Joshi\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta 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