{"id":284,"date":"2014-12-24T07:09:25","date_gmt":"2014-12-24T07:09:25","guid":{"rendered":"https:\/\/delveinsightblog.wordpress.com\/?p=284"},"modified":"2021-07-24T12:56:23","modified_gmt":"2021-07-24T07:26:23","slug":"delveinsights-metabolic-disorders-based-gene-therapy-reports","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/delveinsights-metabolic-disorders-based-gene-therapy-reports","title":{"rendered":"DelveInsight\u2019s Metabolic 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-69e35924e8cc4\" 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-69e35924e8cc4\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-metabolic-disorders-based-gene-therapy-reports\/#Gene_Therapy_to_%E2%80%9Creverse%E2%80%9D_Metabolic_disorders\" >Gene Therapy to \u201creverse\u201d Metabolic disorders!<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-metabolic-disorders-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-3\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-metabolic-disorders-based-gene-therapy-reports\/#Gene_Therapy_Role_in_Metabolic_disorders\" >Gene Therapy: Role in Metabolic disorders<\/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-metabolic-disorders-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-5\" href=\"https:\/\/www.delveinsight.com\/blog\/delveinsights-metabolic-disorders-based-gene-therapy-reports\/#DelveInsights_Gene_Therapy_Reports\" >DelveInsight\u2019s Gene Therapy Reports<\/a><\/li><\/ul><\/nav><\/div>\n<blockquote>\n<h3 style=\"text-align:center;\"><span class=\"ez-toc-section\" id=\"Gene_Therapy_to_%E2%80%9Creverse%E2%80%9D_Metabolic_disorders\"><\/span><span style=\"color:#000080;\">Gene Therapy to \u201creverse\u201d Metabolic disorders!<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<\/blockquote>\n<p style=\"text-align:justify;\"><strong>Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants.<\/strong><\/p>\n<h3><span class=\"ez-toc-section\" id=\"About_Gene_Therapy\"><\/span><span style=\"color:#997640;\">About Gene Therapy<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\">Gene therapy is defined as the treatment or cure of human diseases by transfer of nucleic acids (DNA or RNA) to affected cells and it covers a broad range of therapeutic applications from Mendelian disorders to complex metabolic diseases. Most diseases caused by genetic deficiencies could in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems like that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\">Metabolic disorders refer to different types of medical conditions caused by genetic defects \u2014 most commonly inherited from both parents \u2014 that interfere with the body&#8217;s metabolism. Metabolism is the complex set of chemical reactions that your body uses to maintain life, including energy production. When these chemical processes don&#8217;t work properly due to a hormone or enzyme deficiency, a metabolic disorder occurs. Inherited metabolic disorders fall into different categories, depending on the specific substance and whether it builds up in harmful amounts (because it can&#8217;t be broken down), it&#8217;s too low, or it&#8217;s missing. Metabolic disorders affects millions of people world-wide and is the fourth or fifth leading cause of death in the developed world. Gene therapy is an experimental form of treatment that aims to use the transcriptional machinery of the patient to produce the active factor that exerts the intended therapeutic effect, ideally in a permanent, tissue-specific and manageable way.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Gene_Therapy_Role_in_Metabolic_disorders\"><\/span><span style=\"color:#997640;\">Gene Therapy: Role in Metabolic disorders<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\">Genetic manipulations for altering gene expression pattern <em>in vivo <\/em>with therapeutic outcome, known as gene therapy, offers a fundamentally different approach to pharmacotherapy for inherited as well as acquired metabolic disorders. Current and possible candidates for gene therapy in the field of metabolic disorders include Sanfilippo type A syndrome, Metachromatic Leukodystrophy (MLD), Crigler-Najjar syndrome, Urea cycle disorders and Diabetic Macular Edema etc. because of their poor prognosis.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\">Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transfer technologies. However, interspecies differences in disease biology, vector performance, host\u2013vector interactions and increased size with the associated necessity to functionally correct a greater number of cells to reach the threshold required for therapeutic benefit are acting as biological limitations in this field. Despite these challenges, it is predictable that the gene therapy based strategy in modulating metabolism and treating metabolic disorders will surely impact how we live a healthy life in the near future.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Gene_Therapy_The_Market_Scenario\"><\/span><span style=\"color:#997640;\">Gene Therapy: The Market Scenario<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\">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 GlaxoSmithKline, Pfizer, and Alnylam etc., are operating in the field of metabolic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Lysogene and RegenX Biosciences etc., are driving the new gene therapy research. The industry\u2019s collective pipeline is brimming with 300+ therapies for various therapeutic areas. Gene therapy has the potential of providing a definitive cure for patients with these diseases. The global market opportunities for gene therapy have risen drastically indicating that there\u2019s big money in gene therapy.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"DelveInsights_Gene_Therapy_Reports\"><\/span><span style=\"color:#997640;\">DelveInsight\u2019s Gene Therapy Reports<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#278c8c;\"><strong>DelveInsight\u2019s Gene Therapy Reports<\/strong> cover the entire gene therapy market insights for metabolic 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 to \u201creverse\u201d Metabolic disorders! Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants. About [&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":[173,180,189,209,212,258,356,386,397,398,479,532,558,588],"industry":[17225],"therapeutic_areas":[17235,17240],"class_list":["post-284","post","type-post","status-publish","format-standard","hentry","category-articles","tag-collaborations","tag-competitive-landscape","tag-crigler-najjar-syndrome","tag-delveinsights-gene-therapy-reports","tag-diabetic-macular-edema","tag-gene-therapy","tag-licensing-opportunities","tag-market-trends","tag-metabolic-disorders","tag-metachromatic-leukodystrophy-mld","tag-pipeline-coverage","tag-sanfilippo-type-a-syndrome","tag-technology-assessments","tag-urea-cycle-disorders","industry-pharmaceutical","therapeutic_areas-cell-and-gene-therapy","therapeutic_areas-endocrinology-and-metabolic-disorders"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v25.8 (Yoast SEO v25.8) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Metabolic disorders based Gene Therapy Market Reports | DelveInsight<\/title>\n<meta name=\"description\" content=\"Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. 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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\">collaborations<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Competitive landscape<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Crigler-Najjar syndrome<\/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\">Diabetic Macular Edema<\/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\">market trends<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Metabolic disorders<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Metachromatic Leukodystrophy (MLD)<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Pipeline coverage<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Sanfilippo type A syndrome<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">technology assessments<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Urea cycle disorders<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">collaborations<\/span>","<span class=\"advgb-post-tax-term\">Competitive landscape<\/span>","<span class=\"advgb-post-tax-term\">Crigler-Najjar syndrome<\/span>","<span class=\"advgb-post-tax-term\">DelveInsight&#039;s Gene Therapy Reports<\/span>","<span class=\"advgb-post-tax-term\">Diabetic Macular Edema<\/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\">market trends<\/span>","<span class=\"advgb-post-tax-term\">Metabolic disorders<\/span>","<span class=\"advgb-post-tax-term\">Metachromatic Leukodystrophy (MLD)<\/span>","<span class=\"advgb-post-tax-term\">Pipeline coverage<\/span>","<span class=\"advgb-post-tax-term\">Sanfilippo type A syndrome<\/span>","<span class=\"advgb-post-tax-term\">technology assessments<\/span>","<span class=\"advgb-post-tax-term\">Urea cycle disorders<\/span>"]}},"comment_count":"0","relative_dates":{"created":"Posted 11 years ago","modified":"Updated 5 years ago"},"absolute_dates":{"created":"Posted on Dec 24, 2014","modified":"Updated on Jul 24, 2021"},"absolute_dates_time":{"created":"Posted on Dec 24, 2014 7:09 am","modified":"Updated on Jul 24, 2021 12:56 pm"},"featured_img_caption":"","series_order":"","_links":{"self":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/284","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=284"}],"version-history":[{"count":0,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/284\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media?parent=284"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/categories?post=284"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/tags?post=284"},{"taxonomy":"industry","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/industry?post=284"},{"taxonomy":"therapeutic_areas","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/therapeutic_areas?post=284"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}