{"id":10844,"date":"2021-05-20T12:54:00","date_gmt":"2021-05-20T12:54:00","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=10844"},"modified":"2022-05-03T17:59:00","modified_gmt":"2022-05-03T17:59:00","slug":"endeavor-gene-therapy-for-dmd","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/endeavor-gene-therapy-for-dmd\/","title":{"rendered":"ENDEAVOR, gene therapy for DMD"},"content":{"rendered":"<h2>Sarepta Therapeutics\u2019 investigational gene therapy for the treatment of Duchenne muscular dystrophy, SRP-9001, demonstrates robust expression and consistent safety profile using Sarepta\u2019s commercial process material.<\/h2>\n<p>&nbsp;<\/p>\n<ul type=\"disc\">\n<li><strong>The first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus.<\/strong><\/li>\n<li><strong>Treated patients achieved mean micro-dystrophin expression levels of 55.4% of normal as measured by western blot.<\/strong><\/li>\n<li><strong>Micro-dystrophin was properly localized to the muscle sarcolemma, with patients achieving a mean percentage of dystrophin positive fibres of 70.5% and intensity of micro-dystrophin expression of 116.9% of normal control measured by <\/strong><strong>immunofluorescence<\/strong><strong> (IF).<\/strong><\/li>\n<li><strong>Safety profile consistent with prior studies and no new safety signals identified.<\/strong><\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<p align=\"justify\">Original press release &gt; <span style=\"color: #0000ff;\"><a style=\"color: #0000ff;\" href=\"https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-investigational-gene-therapy-treatment\" target=\"_blank\" rel=\"noopener\">CAMBRIDGE, Mass., May 18, 2021 (GLOBE NEWSWIRE)<\/a><\/span><\/p>\n<p style=\"text-align: left;\"><strong>Sarepta Therapeutics, Inc. announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR conducted in partnership with Roche. In results from the first clinical study using commercially representative material, SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies, supporting its potentially differentiated profile for the treatment of Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.<\/strong><\/p>\n<p>&nbsp;<\/p>\n<blockquote><p><strong>Doug Ingram, president and chief executive officer, Sarepta &#8211;<\/strong> \u201cWe are delighted by these seminal results from the ENDEAVOR Study, our first trial results with SRP-9001 made by our commercial-scale manufacturing process. These data show strong transduction of the micro-dystrophin gene, resulting in robust expression of the properly localized micro-dystrophin protein, and did so with no new or unexpected safety signals. In addition to characterizing and differentiating SRP-9001, these results confirm the extraordinary work done over the last two and a half years to build an at-scale gene therapy manufacturing process and corresponding analytics sufficient to meet the needs of the Duchenne population with what we believe will be a potentially life-changing therapy. Armed with these data, we will seek a meeting with the FDA with the goal of rapidly starting our registrational study.\u201d<\/p><\/blockquote>\n<p>&nbsp;<\/p>\n<h4 style=\"text-align: left;\" align=\"justify\">Observed results<\/h4>\n<p align=\"justify\">In the open-label study, 20 participants between the ages of four and seven were treated with a single infusion of SRP-9001 at a dose of 1.33&#215;10<sup>14<\/sup> VG\/kg. In muscle biopsies from the first 11 patients taken 12 weeks after treatment, the following results were observed:<\/p>\n<ul type=\"disc\">\n<li>All patients demonstrated robust transduction, with a mean micro-dystrophin expression of 55.4% normal, as measured by western blot.<\/li>\n<li>Muscle dystrophin levels demonstrated a mean of 70.5% (baseline 12.8%) muscle fibres expressing micro-dystrophin at 12 weeks with a mean intensity at the sarcolemma of 116.9% (baseline 41.0%) compared to normal biopsies, as measured by immunofluorescence. Comparisons between baseline and post-treatment measures were statistically significant (p=0.001 for positive fibres and p=0.002 for intensity).<\/li>\n<li>Mean vector genome copies per nucleus reached 3.87.<\/li>\n<\/ul>\n<p align=\"justify\"><strong>The safety profile of SRP-9001 observed in the first 11 participants in ENDEAVOR is consistent with the safety seen in earlier studies using clinical manufacturing process material. In line with previously reported clinical data, no clinically relevant complement activation was observed in these 11 patients. Two patients experienced serious adverse events (transaminase elevation in one patient and nausea and vomiting in a second patient) that fully resolved.<\/strong><\/p>\n<h4>About SRP-9001-103 (ENDEAVOR)<\/h4>\n<p>Study SRP-9001-103 (Study 103) is an open-label clinical trial of SRP-9001 that has enrolled 20 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 103 uses commercially representative SRP-9001, and the primary endpoint is the change from baseline in the quantity of micro-dystrophin protein expression measured by western blot at 12 weeks. Secondary outcome measures include change from baseline in micro-dystrophin expression fibre intensity measured by immunofluorescence (IF) and micro-dystrophin expression measured by IF percent dystrophin positive fibres at 12 weeks. Exploratory endpoints include the change in vector genome copies per nucleus, North Star Ambulatory Assessment (NSAA) and certain timed functional tests. Including the initial 12-week period, patients will be followed for a total of five years.<\/p>\n<h4>About SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)<\/h4>\n<p>SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche\u2019s global reach, commercial presence and regulatory expertise with Sarepta\u2019s gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children\u2019s Hospital.<\/p>\n<h4>About Duchenne Muscular Dystrophy<\/h4>\n<p>Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease in approximately one in every 3,500-5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms and other areas. Most patients require full-time use of a wheelchair in their early teens and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.<\/p>\n<h4>About Sarepta Therapeutics<\/h4>\n<p>Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. They hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and they currently have more than 40 programs in various stages of development. Their multi-platform Precision Genetic Medicine Engine drives our vast pipeline in gene therapy, RNA and gene editing. For more information, please visit <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=65fbmY2nP4d98vIpRPLhpfjjCn905WVl0EJEIaI4Td2T177xz_YPZTQtux0XeToaoZxwAAL5XnUv3yKUL_2Uog==\" target=\"_blank\" rel=\"nofollow noopener\"><span style=\"color: #0000ff;\">www.sarepta.com<\/span><\/a> or follow them on <span style=\"color: #0000ff;\"><a style=\"color: #0000ff;\" href=\"https:\/\/www.globenewswire.com\/Tracker?data=tOiC06OBK3gLXUphBAJoPQgxZfBt-a5tERt5J6ApTTVQegb3aGwxbxBffiJgptIl2pX7FcBk37uW6Oih1cG8wg==\" target=\"_blank\" rel=\"nofollow noopener\">Twitter<\/a>,\u00a0<a style=\"color: #0000ff;\" href=\"https:\/\/www.globenewswire.com\/Tracker?data=FO4SE53jx10KK4fjojC8xLFRPNNSfuuj4F87lYhNs6mJQw3V_Ky_WOeC4a9Ucsj1v4c2QSAls_eWJ2pPrq0I6OLY5i1YIZEnOfRo5NBIR3QLrv8zG47NfZ_O7rixaLiT\" target=\"_blank\" rel=\"nofollow noopener\">LinkedIn<\/a>,\u00a0<a style=\"color: #0000ff;\" href=\"https:\/\/www.globenewswire.com\/Tracker?data=881xarbHHnl6lOnSGgPoEBHdQB7zfJccQOkd2aVs4Cpcmx1zNAapjxDSfILtzTFPN9BHzw74mWfBmatACxpBOgw-9o0ROA-SfW2XTgJW7ew=\" target=\"_blank\" rel=\"nofollow noopener\">Instagram<\/a><\/span>\u00a0and<span style=\"color: #0000ff;\">\u00a0<a style=\"color: #0000ff;\" href=\"https:\/\/www.globenewswire.com\/Tracker?data=Nb0aS-gDRqRVsjfDMgLSVl1k7A5ONoEjs3ZqmrUnTlhg5fUJT5X5tQRmQ2-lmS1rRGjKPyhHAUNQUBGllpggq0UtiHDOx7guEdmJUxQzQBP5lOCEv9W4Wr2Y2c8dXmBg\" target=\"_blank\" rel=\"nofollow noopener\">Facebook<\/a><\/span>.<\/p>\n<h4>More interesting links<\/h4>\n<ul>\n<li><a href=\"https:\/\/www.sarepta.com\/our-pipeline\" target=\"_blank\" rel=\"noopener noreferrer\"><span style=\"color: #0000ff;\">Sarepta drug pipeline<\/span><\/a><\/li>\n<li><span style=\"color: #0000ff;\"><a style=\"color: #0000ff;\" href=\"\/?page_id=8990\" target=\"_blank\" rel=\"noopener noreferrer\">Grounded in the DMD community<\/a><\/span><\/li>\n<li><span style=\"color: #0000ff;\"><a style=\"color: #0000ff;\" href=\"https:\/\/www.sarepta.com\/\" rel=\"noopener\">Sarepta Therapeutics, Inc.<\/a><\/span><\/li>\n<li><a href=\"https:\/\/musculardystrophynews.com\/raavrh74-mhck7-micro-dystrophin\/\" target=\"_blank\" rel=\"noopener noreferrer\"><span style=\"color: #0000ff;\">SRP-9001 Micro-Dystrophin<\/span>\u00a0<\/a>from\u00a0Muscular Dystrophy News Today<\/li>\n<li><a href=\"https:\/\/musculardystrophynews.com\/gene-therapy-in-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\"><span style=\"color: #0000ff;\">Gene Therapy<\/span><\/a>\u00a0from\u00a0Muscular Dystrophy News Today<\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"<p>Sarepta Therapeutics\u2019 investigational gene therapy for the treatment of Duchenne muscular dystrophy, SRP-9001, demonstrates robust expression and consistent safety profile using Sarepta\u2019s commercial process material. &nbsp; The first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus. Treated patients achieved mean micro-dystrophin expression levels of [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":10859,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[12,17,18],"tags":[],"class_list":["post-10844","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-research","category-treatments"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>ENDEAVOR, gene therapy for DMD - La Force dmd<\/title>\n<meta name=\"description\" content=\"Sarepta announced positive results from the Study SRP-9001-103, an open-label study known as ENDEAVOR conducted in partnership with Roche.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/laforcedmd.com\/en\/endeavor-gene-therapy-for-dmd\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"ENDEAVOR, gene therapy for DMD - 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