{"id":226,"date":"2014-12-20T09:41:12","date_gmt":"2014-12-20T09:41:12","guid":{"rendered":"https:\/\/delveinsightblog.wordpress.com\/?p=226"},"modified":"2025-05-05T18:21:07","modified_gmt":"2025-05-05T12:51:07","slug":"delveinsights-central-nervous-system-based-gene-therapy-reports","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports","title":{"rendered":"DelveInsight\u2019s Central Nervous System Disorders based Gene Therapy Reports"},"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-69f2e7ab2f1ff\" 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-69f2e7ab2f1ff\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><ul class='ez-toc-list-level-4' ><li class='ez-toc-heading-level-4'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#Gene_Therapy_The_%E2%80%9CHunt%E2%80%9D_for_CNS_disorders\" >Gene Therapy: The \u201cHunt\u201d for CNS disorders!<\/a><ul class='ez-toc-list-level-5' ><li class='ez-toc-heading-level-5'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#Many_companies_have_tried_over_the_years_to_find_a_way_to_reverse_the_course_of_crippling_central_nervous_system_disorders_like_amyotrophic_lateral_sclerosis_aka_Lou_Gehrigs_disease_but_have_failed_Fortunately_Gene_therapy_has_proven_to_be_an_answer_to_a_wide_range_of_serious_central_nervous_system_disorders_like_Lou_Gehrigs_disease_Friedreichs_Ataxia_and_Parkinsons_etc\" >Many companies have tried over the years to find a way to reverse the course of crippling central nervous system disorders like amyotrophic lateral sclerosis (a.k.a. Lou Gehrig\u2019s disease) but have failed. Fortunately, Gene therapy has proven to be an answer to a wide range of serious central nervous system disorders like Lou Gehrig\u2019s disease, Friedreich\u2019s Ataxia, and Parkinson\u2019s etc.<\/a><\/li><\/ul><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#About_Gene_Therapy\" >About Gene Therapy<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#Gene_Therapy_Role_in_CNS\" >Gene Therapy: Role in CNS<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#Gene_Therapy_The_Market_Scenario\" >Gene Therapy: The Market Scenario<\/a><\/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\/delveinsights-central-nervous-system-based-gene-therapy-reports\/#DelveInsights_Gene_Therapy_Reports\" >DelveInsight\u2019s Gene Therapy Reports<\/a><\/li><\/ul><\/nav><\/div>\n<blockquote>\n<h4 style=\"text-align:center;\"><span class=\"ez-toc-section\" id=\"Gene_Therapy_The_%E2%80%9CHunt%E2%80%9D_for_CNS_disorders\"><\/span><span style=\"color:#800000;\">Gene Therapy: The \u201cHunt\u201d for CNS disorders!<\/span><span class=\"ez-toc-section-end\"><\/span><\/h4>\n<\/blockquote>\n<h5 style=\"text-align:justify;\"><span class=\"ez-toc-section\" id=\"Many_companies_have_tried_over_the_years_to_find_a_way_to_reverse_the_course_of_crippling_central_nervous_system_disorders_like_amyotrophic_lateral_sclerosis_aka_Lou_Gehrigs_disease_but_have_failed_Fortunately_Gene_therapy_has_proven_to_be_an_answer_to_a_wide_range_of_serious_central_nervous_system_disorders_like_Lou_Gehrigs_disease_Friedreichs_Ataxia_and_Parkinsons_etc\"><\/span><strong>Many companies have tried over the years to find a way to reverse the course of crippling central nervous system disorders like amyotrophic lateral sclerosis (a.k.a. Lou Gehrig\u2019s disease) but have failed. Fortunately, Gene therapy has proven to be an answer to a wide range of serious central nervous system disorders like Lou Gehrig\u2019s disease, Friedreich\u2019s Ataxia, and Parkinson\u2019s etc.<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h5>\n<h3><span class=\"ez-toc-section\" id=\"About_Gene_Therapy\"><\/span><span style=\"color:#000080;\">About Gene Therapy<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#333300;\">Gene therapy involves the use of a modified virus\u2014a viral vector\u2014as a vehicle to deliver healthy genes into a cell to replace faulty or missing ones. This is a new method with potential for treating a broad range of acquired and inherited neurologic diseases, where the causative gene defect or deletion has been identified. In addition to gene replacement the application of gene products that reduce cellular dysfunction or death represent new therapeutic options. Inspite of the significant setbacks and limited success that the field of gene therapy has experienced, it has the potential to succeed where so many drugs have failed by providing a way to either cure, or significantly reverse the effects of these enervating diseases.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Gene_Therapy_Role_in_CNS\"><\/span><span style=\"color:#000080;\">Gene Therapy: Role in CNS<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#333300;\">The potential of Gene therapy in the field of Central Nervous System disorders has been propelled by genetic technologies, including identifying disease genes and disruptive mutations, design of genomic interacting elements to regulate transcription etc. These versatile new tools for manipulation of genetic elements provide the ability to tailor the mode of genetic intervention to specific aspects of a disease state.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#333300;\">The reasons for Central Nervous System based disorders to become a preferred area of application for gene therapy is the number of rare genetic disorders affecting the brain that don\u2019t have any effective treatments. Moreover, a variety of gene delivery methods now provide strong scientific underpinnings for the design and implementation of powerful therapeutic strategies that can tackle focal as well as global disease. Additionally, nervous system offers several practical advantages for intervention, including a reduced immune response to vectors and transgenes in the brain parenchyma as compared with other peripheral sites of injection, the presence of only a few normal replicating cells that might be transformed by vector integration into their genome, and the reduced ability of vectors injected into the brain to access germline tissues as compared with peripheral injections. However, insufficient resources and funds are proving as a hurdle resulting in marginal success in this field. Inspite of this, there is much optimism and confidence in the potential of Gene therapy to develop an effective treatment for neurologic diseases in the near future.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Gene_Therapy_The_Market_Scenario\"><\/span><span style=\"color:#333399;\">Gene Therapy: The Market Scenario<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#333300;\">Many companies are investing in and researching on this field using gene therapy due to its promising effects. Large Pharmaceutical and Biotech giants, such as UniQure Biopharma, Sanofi, Oxford BioMedica etc., are operating in the field of Central Nervous System disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Audentes and Genethon etc., are driving the new gene therapy research. The industry\u2019s collective pipeline is brimming with 300+ therapies of which CNS collectively accounts for 12 % of gene therapies in pipeline. The global market opportunities for gene therapy have risen drastically and the tide has been turning back to enthusiastic support.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"DelveInsights_Gene_Therapy_Reports\"><\/span><span style=\"color:#333399;\">DelveInsight\u2019s Gene Therapy Reports<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#3366ff;\"><strong>DelveInsight\u2019s Gene Therapy Reports<\/strong><\/span> <span style=\"color:#333300;\">cover the entire gene therapy market insights for central nervous system based disorders including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight\u2019s proprietary market and pipeline analytics which identifies the front runners in this therapeutic area. It also identifies the potential market movers and future regulatory landscape.<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Gene Therapy: The \u201cHunt\u201d for CNS disorders! Many companies have tried over the years to find a way to reverse the course of crippling central nervous system disorders like amyotrophic lateral sclerosis (a.k.a. Lou Gehrig\u2019s disease) but have failed. Fortunately, Gene therapy has proven to be an answer to a wide range of serious central [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","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":[74,158,171,173,209,251,258,356,362,386,419,450,480,558],"industry":[17225],"therapeutic_areas":[17235,17229],"class_list":["post-226","post","type-post","status-publish","format-standard","hentry","category-articles","tag-amyotrophic-lateral-sclerosis","tag-central-nervous-system","tag-cns","tag-collaborations","tag-delveinsights-gene-therapy-reports","tag-friedreichs-ataxia","tag-gene-therapy","tag-licensing-opportunities","tag-lou-gehrigs-disease","tag-market-trends","tag-neurologic-diseases","tag-parkinsons","tag-pipeline-coverage-and-competitive-landscape","tag-technology-assessments","industry-pharmaceutical","therapeutic_areas-cell-and-gene-therapy","therapeutic_areas-central-nervous-system"],"acf":[],"yoast_head":"<!-- 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href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Articles<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">Articles<\/span>"]},"tags":{"linked":["<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">amyotrophic lateral sclerosis<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Central nervous system<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">CNS<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">collaborations<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">DelveInsight&#039;s Gene Therapy Reports<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Friedreich\u2019s Ataxia<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Gene therapy<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">licensing opportunities<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Lou Gehrig\u2019s disease<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">market trends<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">neurologic diseases<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Parkinson\u2019s<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">pipeline coverage and competitive landscape<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">technology assessments<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">amyotrophic lateral sclerosis<\/span>","<span class=\"advgb-post-tax-term\">Central nervous system<\/span>","<span class=\"advgb-post-tax-term\">CNS<\/span>","<span class=\"advgb-post-tax-term\">collaborations<\/span>","<span class=\"advgb-post-tax-term\">DelveInsight&#039;s Gene Therapy Reports<\/span>","<span class=\"advgb-post-tax-term\">Friedreich\u2019s Ataxia<\/span>","<span class=\"advgb-post-tax-term\">Gene therapy<\/span>","<span class=\"advgb-post-tax-term\">licensing opportunities<\/span>","<span class=\"advgb-post-tax-term\">Lou Gehrig\u2019s disease<\/span>","<span class=\"advgb-post-tax-term\">market trends<\/span>","<span class=\"advgb-post-tax-term\">neurologic diseases<\/span>","<span class=\"advgb-post-tax-term\">Parkinson\u2019s<\/span>","<span class=\"advgb-post-tax-term\">pipeline coverage and competitive landscape<\/span>","<span class=\"advgb-post-tax-term\">technology assessments<\/span>"]}},"comment_count":"0","relative_dates":{"created":"Posted 11 years ago","modified":"Updated 12 months ago"},"absolute_dates":{"created":"Posted on Dec 20, 2014","modified":"Updated on May 5, 2025"},"absolute_dates_time":{"created":"Posted on Dec 20, 2014 9:41 am","modified":"Updated on May 5, 2025 6:21 pm"},"featured_img_caption":"","series_order":"","_links":{"self":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/226","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/comments?post=226"}],"version-history":[{"count":1,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/226\/revisions"}],"predecessor-version":[{"id":31689,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/226\/revisions\/31689"}],"wp:attachment":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media?parent=226"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/categories?post=226"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/tags?post=226"},{"taxonomy":"industry","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/industry?post=226"},{"taxonomy":"therapeutic_areas","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/therapeutic_areas?post=226"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}