{"id":17818,"date":"2024-12-13T12:25:36","date_gmt":"2024-12-13T04:25:36","guid":{"rendered":"https:\/\/flcube.com\/?p=17818"},"modified":"2024-12-13T12:50:27","modified_gmt":"2024-12-13T04:50:27","slug":"huidagene-therapeutics-launches-muscle-study-for-crispr-therapy-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=17818","title":{"rendered":"HuidaGene Therapeutics Launches MUSCLE Study for CRISPR Therapy in Duchenne Muscular Dystrophy"},"content":{"rendered":"\n<p>Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for its CRISPR-based DNA-editing therapy, HG302, which targets Duchenne muscular dystrophy (DMD), with the first subject now dosed.<\/p>\n\n\n\n<p><strong>Understanding Duchenne Muscular Dystrophy and HG302<\/strong><br>Duchenne muscular dystrophy is a severe X-linked recessive disorder and a prevalent form of progressive muscular dystrophy. It is caused by mutations in the DMD gene, leading to the absence of functional dystrophin protein. The disease&#8217;s progression results in increasing muscle weakness, muscle degeneration, loss of ambulation by adolescence, life-threatening complications in early adulthood, and a shortened lifespan. Current treatment options are limited to symptom management. HG302, utilizing HuidaGene\u2019s proprietary hfCas12Max, a high-fidelity CRISPR nuclease developed through the AI-guided HG-PRECISE platform, precisely edits the DMD exon51 splice-donor site, enabling exon skipping to restore dystrophin production. This one-time treatment option for DMD patients amenable to exon51 skipping or reframing has previously obtained Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) in the US.<\/p>\n\n\n\n<p><strong>MUSCLE Trial Design and Objectives<\/strong><br>The MUSCLE (Multidose study to Understand the Safety of Crispr editing therapy and its long-Lasting Effects in DMD) trial is an open-label, multidose dose-escalation, first-in-human study. It is expected to enroll ambulatory boys between 4 and 8 years old with DMD who exhibit impaired muscle function. The study aims to evaluate the safety, tolerability, and preliminary efficacy of HG302. Key outcomes include dystrophin restoration, functional muscle improvements, and quality of life metrics.<a href=\"https:\/\/flcube.com\">-Fineline Info &amp; Tech<\/a><\/p>\n\n\n\n<p><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for&#8230;<\/p>\n","protected":false},"author":1,"featured_media":17820,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"googlesitekit_rrm_CAownpewDA:productID":"","_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_publicize_message":"","jetpack_publicize_feature_enabled":true,"jetpack_social_post_already_shared":false,"jetpack_social_options":{"image_generator_settings":{"template":"highway","default_image_id":0,"font":"","enabled":false},"version":2}},"categories":[7,11],"tags":[62,1619,24],"class_list":["post-17818","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-company","category-drug","tag-clinical-trial-approval-initiation","tag-huidagene-therapeutics","tag-rare-orphan-disease-drugs"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v23.6 (Yoast SEO v27.5) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>HuidaGene Therapeutics Launches MUSCLE Study for CRISPR Therapy in Duchenne Muscular Dystrophy - Insight, China&#039;s Pharmaceutical Industry<\/title>\n<meta name=\"description\" content=\"Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for its CRISPR-based DNA-editing therapy, HG302, which targets Duchenne muscular dystrophy (DMD), with the first subject now dosed.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/flcube.com\/?p=17818\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"HuidaGene Therapeutics Launches MUSCLE Study for CRISPR Therapy in Duchenne Muscular Dystrophy\" \/>\n<meta property=\"og:description\" content=\"Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for its CRISPR-based DNA-editing therapy, HG302, which targets Duchenne muscular dystrophy (DMD), with the first subject now dosed.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/flcube.com\/?p=17818\" \/>\n<meta property=\"og:site_name\" content=\"Insight, China&#039;s Pharmaceutical Industry\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/profile.php?id=61566606313834\" \/>\n<meta property=\"article:published_time\" content=\"2024-12-13T04:25:36+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2024-12-13T04:50:27+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/flcube.com\/wp-content\/uploads\/2024\/12\/1304.png\" \/>\n\t<meta property=\"og:image:width\" content=\"1080\" \/>\n\t<meta property=\"og:image:height\" content=\"721\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"Fineline Cube\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:description\" content=\"Navigate the complexities of China\u2019s pharmaceutical industry with precision and clarity. 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