{"id":10492,"date":"2019-10-25T12:59:33","date_gmt":"2019-10-25T07:29:33","guid":{"rendered":"https:\/\/www.rootsanalysis.com\/blog\/?p=10492"},"modified":"2026-07-27T13:14:44","modified_gmt":"2026-07-27T07:44:44","slug":"gene-therapy-pipeline-update-orchard-therapeutics-presents-data-on-metachromatic-leukodystrophy","status":"publish","type":"post","link":"https:\/\/www.rootsanalysis.com\/blog\/gene-therapy-pipeline-update-orchard-therapeutics-presents-data-on-metachromatic-leukodystrophy\/","title":{"rendered":"Gene Therapy Pipeline Update: Orchard Therapeutics Presents Data on Metachromatic Leukodystrophy"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Orchard Therapeutics, a commercial-stage biopharmaceutical company,&nbsp;announced initial results from a clinical trial (<a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03392987\">NCT03392987<\/a>) with a cryopreserved formulation of OTL-200, gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). Get the details of the results <a href=\"https:\/\/ir.orchard-tx.com\/news-releases\/news-release-details\/orchard-therapeutics-presents-data-otl-200-patients\">here<\/a>.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>What is OTL-200<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><span class=\"hit_org\">OTL-200<\/span>\u00a0is autologous CD34+ cells transduced with a lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA) used for the treatment of MLD.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>What is\u00a0metachromatic leukodystrophy (MLD)<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system.\u00a0This condition is called <a href=\"https:\/\/rarediseases.org\/rare-diseases\/metachromatic-leukodystrophy\/\">metachromatic leukodystrophy<\/a> because when viewed under a microscope, sulfatide accumulation in cells appears as granules that are colored differently than other cellular material (metachromatic).<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>What is the prevalence of metachromatic leukodystrophy (MLD)<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">The true prevalence rate of MLD is unknown, but is <a href=\"https:\/\/rarediseases.org\/rare-diseases\/metachromatic-leukodystrophy\/\">estimated<\/a> to be between 1 in 40,000 and 1 in 160,000. The Navajo also have a higher prevalence rate of 1 in every 2,500 people. In certain populations in the Middle East, these numbers may be even higher.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Since the drug is being developed for a rare disease, has it received any designations:&nbsp;<\/strong>The drug has received two designations:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Orphan Drug Designation (2007, EMA, MLD)<\/li>\n\n\n\n<li><a href=\"https:\/\/www.b3cnewswire.com\/201805031758\/orchard-therapeutics-otl-200-receives-rare-pediatric-disease-designation-from-fda-for-treatment-of-metachromatic-leukodystrophy.html\">Rare Pediatric Disease Designation (USFDA, 2018, MLD)<\/a><\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>What is the overall gene therapy pipeline like for metachromatic leukodystrophy (MLD)<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">As per a recent report by Roots Analysis, there are not many gene therapies that are currently being developed for MLD.<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"517\" src=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Pipeline-for-Metachromatic-Leukodystrophy-Roots-Analysis-1024x517.webp\" alt=\"Infographic: MLD Gene Therapy Pipeline for metachromatic leukodystrophy (MLD) as per analysis done by Roots Analysis\" class=\"wp-image-17697\" style=\"object-fit:contain;width:800px;height:405px\" srcset=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Pipeline-for-Metachromatic-Leukodystrophy-Roots-Analysis-1024x517.webp 1024w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Pipeline-for-Metachromatic-Leukodystrophy-Roots-Analysis-300x151.webp 300w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Pipeline-for-Metachromatic-Leukodystrophy-Roots-Analysis-768x388.webp 768w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Pipeline-for-Metachromatic-Leukodystrophy-Roots-Analysis.webp 1373w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">To get more insights about the gene therapy pipeline, check out the detailed report <a href=\"https:\/\/www.rootsanalysis.com\/reports\/gene-therapies-market\/268.html\">Gene Therapy Market<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Orchard Therapeutics, a commercial-stage biopharmaceutical company,&nbsp;announced initial results from a clinical trial (NCT03392987) with a cryopreserved formulation of OTL-200, gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). Get the details of the results here. What is OTL-200 OTL-200\u00a0is autologous CD34+ cells transduced with a lentiviral vector containing human arylsulfatase A (ARSA) complementary [&hellip;]<\/p>\n","protected":false},"author":21,"featured_media":17270,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[47],"tags":[48,329],"class_list":["post-10492","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-gene-therapy","tag-gene-therapy","tag-therapeutics"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene Therapy Pipeline Update: Orchard Therapeutics.. | Blog<\/title>\n<meta name=\"description\" content=\"As per a recent report by Roots Analysis, there are not many gene Therapy that are currently being developed for MLD.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, 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