{"id":10432,"date":"2019-10-22T21:31:03","date_gmt":"2019-10-22T16:01:03","guid":{"rendered":"https:\/\/www.rootsanalysis.com\/blog\/?p=10432"},"modified":"2025-05-08T15:35:41","modified_gmt":"2025-05-08T10:05:41","slug":"gene-therapy-pipeline-update-rocket-pharmaceuticals-receives-ind-clearance-for-gene-therapy-for-pyruvate-kinase-deficiency","status":"publish","type":"post","link":"https:\/\/www.rootsanalysis.com\/blog\/gene-therapy-pipeline-update-rocket-pharmaceuticals-receives-ind-clearance-for-gene-therapy-for-pyruvate-kinase-deficiency\/","title":{"rendered":"Gene Therapy Pipeline Update: Rocket Pharmaceuticals Receives IND Clearance for Gene Therapy for Pyruvate Kinase Deficiency"},"content":{"rendered":"<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"768\" height=\"307\" src=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Rocket-Pharmaceuticals-Logo-Roots.webp\" alt=\"Representation only logo for Rocket Pharma\" class=\"wp-image-17700\" style=\"object-fit:contain;width:500px;height:200px\" srcset=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Rocket-Pharmaceuticals-Logo-Roots.webp 768w, https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2023\/10\/Rocket-Pharmaceuticals-Logo-Roots-300x120.webp 300w\" sizes=\"auto, (max-width: 768px) 100vw, 768px\" \/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/rocketpharma.com\/our-disease-focus\/\">Rocket Pharmaceuticals<\/a>, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating inherited diseases announced clearance of Investigational New Drug (IND) by the FDA for RP-L301 gene therapy. RP-L301 is a\u00a0lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a genetic rare disease. Last month, the company received the\u00a0Investigational Medicinal Product Dossier (IMPD) clearance from the Spanish Agency for Medicines and Health Products (AEMPS) for RP-L301<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">According to the company&#8217;s CEO,&nbsp;<em>&#8220;RP-L301 represents the first gene therapy candidate in development for PKD, a hematologic disorder in which the current treatment options, chronic blood transfusions, and splenectomy, are associated with burdensome side effects and end-organ damage.&#8221;<\/em><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Check out the full news <a href=\"https:\/\/rocketpharmaceuticals.gcs-web.com\/news-releases\/news-release-details\/rocket-pharmaceuticals-announces-clearance-ind-rp-l301-gene\">here<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Last month, the company registered a phase I clinical trial (NCT04105166), for which the recruitments are yet to begin. The drug is being developed for <a href=\"https:\/\/rarediseases.org\/rare-diseases\/pyruvate-kinase-deficiency\/\">PKD deficiency<\/a>, a genetic disorder, which is caused by a mutation in the <em>PKLR<\/em>&nbsp;gene. This gene contains instructions for creating a specialized protein (enzyme) known as pyruvate kinase, which is vital for the breakdown of adenosine triphosphate (<a href=\"https:\/\/www.sciencedirect.com\/topics\/neuroscience\/adenosine-triphosphate\">ATP<\/a>) &#8211; the energy-carrying-molecule.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Over the last few years, gene therapies have proven to be effective tools in curing genetic disorders. Yesterday, Homology Medicines, a company using proprietary gene editing and gene therapy platform&nbsp;announced&nbsp;the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of&nbsp;<a href=\"https:\/\/rarediseases.org\/rare-diseases\/metachromatic-leukodystrophy\/\">metachromatic leukodystrophy<\/a>&nbsp;(MLD) and&nbsp;phenylketonuria&nbsp;(PKU).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The ongoing <a href=\"https:\/\/www.statnews.com\/2019\/10\/22\/novartis-gene-therapy-brings-in-160-million-beating-expectations\/\">success ride<\/a> of the approved gene therapies has increased expectations in the overall gene therapy market, which is expected to be around $12 billion by 2030.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Check out some of the free insights <a href=\"https:\/\/www.rootsanalysis.com\/blog\/gene-therapy-market-a-strong-pipeline-with-12-billion-opportunity-by-2030\/\">Gene Therapy Market<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Rocket Pharmaceuticals, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating inherited diseases announced clearance of Investigational New Drug (IND) by the FDA for RP-L301 gene therapy. RP-L301 is a\u00a0lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a genetic rare disease. Last month, the company received [&hellip;]<\/p>\n","protected":false},"author":21,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[47],"tags":[48,65],"class_list":["post-10432","post","type-post","status-publish","format-standard","hentry","category-gene-therapy","tag-gene-therapy","tag-pharmaceuticals"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene Therapy Pipeline Update: Rocket Pharmaceuticals.. | Blog<\/title>\n<meta name=\"description\" content=\"Rocket Pharmaceuticals, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating..\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" 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