{"id":10428,"date":"2019-10-22T20:06:10","date_gmt":"2019-10-22T14:36:10","guid":{"rendered":"https:\/\/www.rootsanalysis.com\/blog\/?p=10428"},"modified":"2026-07-27T13:16:43","modified_gmt":"2026-07-27T07:46:43","slug":"gene-therapy-pipeline-update-homology-medicines-presents-data-demonstrating-preclinical-proof-of-concept","status":"publish","type":"post","link":"https:\/\/www.rootsanalysis.com\/blog\/gene-therapy-pipeline-update-homology-medicines-presents-data-demonstrating-preclinical-proof-of-concept\/","title":{"rendered":"Gene Therapy Pipeline Update: Homology Medicines Presents Data Demonstrating Preclinical Proof-of-Concept"},"content":{"rendered":"<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"251\" src=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-1024x251.webp\" alt=\"logo of homology medicines \" class=\"wp-image-17693\" style=\"object-fit:contain;width:600px;height:148px\" srcset=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-1024x251.webp 1024w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-300x73.webp 300w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-768x188.webp 768w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-1536x376.webp 1536w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Homology-Medicines-Logo-Roots-2048x501.webp 2048w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of <a href=\"https:\/\/rarediseases.org\/rare-diseases\/metachromatic-leukodystrophy\/\">metachromatic leukodystrophy<\/a> (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The presented preclinical data showed the results from studies conducted in the murine model and non-human primates. The results demonstrated the potential of&nbsp;HMI-202 gene therapy in crossing&nbsp;the blood-brain-barrier and the blood-nerve-barrier. In addition, the results also indicated the&nbsp;increased levels of arylsulfatase a (ARSA) protein to therapeutic levels.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Check out the complete results&nbsp;here.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Earlier this year, the company received <a href=\"https:\/\/www.fda.gov\/patients\/fast-track-breakthrough-therapy-accelerated-approval-priority-review\/fast-track\">fast track designation<\/a> for its gene therapy development candidate, HMI-102, which is being developed for adults with PKU. In June, the company also initiated a phase 1\/2 pheNIX trial (NCT03952156) for HMI-102.&nbsp; The company went public in <a href=\"http:\/\/investors.homologymedicines.com\/news-releases\/news-release-details\/homology-medicines-announces-closing-initial-public-offering\">April<\/a>, last year.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Gene Therapies Market<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">The results show positive signs for the future development of gene therapies in patients with genetic diseases and metabolic disorders. The pipeline already has several candidates in development for these therapeutic areas. Here is a snapshot of the gene therapies pipeline.<\/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=\"515\" src=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Development-Pipeline-Roots-3-1024x515.webp\" alt=\"Infographic Gene Therapy Pipeline Review by Therapeutic area and phase of development based on the data collected by Roots analysis\" class=\"wp-image-17695\" style=\"object-fit:contain;width:800px;height:404px\" srcset=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Development-Pipeline-Roots-3-1024x515.webp 1024w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Development-Pipeline-Roots-3-300x151.webp 300w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Development-Pipeline-Roots-3-768x386.webp 768w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Gene-Therapy-Development-Pipeline-Roots-3.webp 1477w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">The overall gene therapy market is expected to reach close to <a href=\"https:\/\/www.rootsanalysis.com\/blog\/gene-therapy-market-a-strong-pipeline-with-12-billion-opportunity-by-2030\/\">$12 billion by 2030<\/a>. More such positive results are expected to drive the growth in this innovative space.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">For detailed insights about the gene therapies market, check out the report Homology Medicines <a href=\"https:\/\/www.rootsanalysis.com\/reports\/gene-therapies-market\/268.html\">Gene Therapy market report<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting. The presented preclinical data showed the results from studies conducted in [&hellip;]<\/p>\n","protected":false},"author":21,"featured_media":17311,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[47],"tags":[48,370],"class_list":["post-10428","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-gene-therapy","tag-gene-therapy","tag-homology-medicines"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Homology Medicines Announces Pre-clinical Data Supporting<\/title>\n<meta name=\"description\" content=\"Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link 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