{"id":19549,"date":"2023-04-18T23:19:00","date_gmt":"2023-04-18T15:19:00","guid":{"rendered":"https:\/\/flcube.com\/?p=19549"},"modified":"2024-12-23T23:21:44","modified_gmt":"2024-12-23T15:21:44","slug":"huidagene-receives-cde-ind-approval-for-ophthalmology-gene-therapy-hg004","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=19549","title":{"rendered":"HuidaGene Receives CDE IND Approval for Ophthalmology Gene Therapy HG004"},"content":{"rendered":"\n<p>China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced receiving Investigational New Drug (IND) approval from the Center for Drug Evaluation (CDE) in China for its ophthalmology gene therapy, HG004. This gene replacement therapy drug utilizes a recombinant non-adeno-associated virus serotype 2 (non-AAV2) vector to deliver a functional human RPE65 gene to the retina, aiming to restore, treat, and prevent blindness in children and adults with RPE65 mutation-associated hereditary retinal dystrophy (IRD).<\/p>\n\n\n\n<p><strong>Study Design and Endpoints<\/strong><br>The multi-country, multi-center, multi-cohort, dosage exploration study is designed to assess the safety, tolerability, efficacy, and long-term clinical persistence of HG004 in RPE65 retinopathy. Primary endpoints include adverse events, specific laboratory tests, and ophthalmic examinations. The multi-luminance mobility testing (MLMT) will also be used to evaluate visual function, providing a comprehensive assessment of the therapy&#8217;s impact on patients&#8217; visual capabilities.<\/p>\n\n\n\n<p><strong>Understanding Hereditary Retinal Dystrophy (IRD)<\/strong><br>IRD is a rare blinding disease caused by genetic mutations, with over 250 reported pathogenic genes. Mutations in the RPE65 gene can lead to a range of conditions including Leber congenital amaurosis (LCA), severe early-onset childhood retinal dystrophy (SECORD), early onset severe retinal dystrophy (EOSRD), or retinitis pigmentosa (RP), all of which are considered RPE65 related retinopathy and represent a phenotype continuum of the same disease. RPE65 gene mutation-associated retinopathy typically manifests between birth and 5 years old, with main clinical manifestations including night blindness, progressive visual field loss, and central visual loss.<a href=\"https:\/\/flcube.com\">-Fineline Info &amp; Tech<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced receiving Investigational New Drug (IND) approval from&#8230;<\/p>\n","protected":false},"author":1,"featured_media":0,"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":[62,66,2091,1597,44],"class_list":["post-19549","post","type-post","status-publish","format-standard","hentry","category-company","category-drug","tag-clinical-trial-approval-initiation","tag-gene-therapy","tag-huidagene-biotechnology","tag-mrct","tag-ophthalmology"],"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 Receives CDE IND Approval for Ophthalmology Gene Therapy HG004 - Insight, China&#039;s Pharmaceutical Industry<\/title>\n<meta name=\"description\" content=\"China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced receiving Investigational New Drug (IND) approval from the Center for Drug Evaluation (CDE) in China for its ophthalmology gene therapy, HG004. This gene replacement therapy drug utilizes a recombinant non-adeno-associated virus serotype 2 (non-AAV2) vector to deliver a functional human RPE65 gene to the retina, aiming to restore, treat, and prevent blindness in children and adults with RPE65 mutation-associated hereditary retinal dystrophy (IRD).\" \/>\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=19549\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"HuidaGene Receives CDE IND Approval for Ophthalmology Gene Therapy HG004\" \/>\n<meta property=\"og:description\" content=\"Navigate the complexities of China\u2019s pharmaceutical industry with precision and clarity. FLCUBE delivers reliable data, news, and intelligence to broaden your horizons and sharpen your decision-making. 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This gene replacement therapy drug utilizes a recombinant non-adeno-associated virus serotype 2 (non-AAV2) vector to deliver a functional human RPE65 gene to the retina, aiming to restore, treat, and prevent blindness in children and adults with RPE65 mutation-associated hereditary retinal dystrophy (IRD).\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=19549#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/flcube.com\\\/?p=19549\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=19549#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/flcube.com\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"HuidaGene Receives CDE IND Approval for Ophthalmology Gene Therapy HG004\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/flcube.com\\\/#website\",\"url\":\"https:\\\/\\\/flcube.com\\\/\",\"name\":\"Insight, China's Pharmaceutical Industry\",\"description\":\"Fineline Insights, Pharma Clarity\",\"publisher\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/flcube.com\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/flcube.com\\\/#organization\",\"name\":\"Fineline Infomation and Technology\",\"alternateName\":\"Fineline Info & Tech\",\"url\":\"https:\\\/\\\/flcube.com\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/flcube.com\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/flcube.com\\\/wp-content\\\/uploads\\\/2024\\\/11\\\/Fineline-info-tech-scaled.jpg\",\"contentUrl\":\"https:\\\/\\\/flcube.com\\\/wp-content\\\/uploads\\\/2024\\\/11\\\/Fineline-info-tech-scaled.jpg\",\"width\":2560,\"height\":1894,\"caption\":\"Fineline Infomation and Technology\"},\"image\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/#\\\/schema\\\/logo\\\/image\\\/\"},\"sameAs\":[\"https:\\\/\\\/www.facebook.com\\\/profile.php?id=61566606313834\"]},{\"@type\":\"Person\",\"@id\":\"https:\\\/\\\/flcube.com\\\/#\\\/schema\\\/person\\\/19ad11870d326204db8524d3c3c5e66a\",\"name\":\"Fineline Cube\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/89f733aaed3d2d91d7d6f45d0e9ab509e3004e8243c6e57620b36380684657f2?s=96&d=mm&r=g\",\"url\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/89f733aaed3d2d91d7d6f45d0e9ab509e3004e8243c6e57620b36380684657f2?s=96&d=mm&r=g\",\"contentUrl\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/89f733aaed3d2d91d7d6f45d0e9ab509e3004e8243c6e57620b36380684657f2?s=96&d=mm&r=g\",\"caption\":\"Fineline Cube\"},\"sameAs\":[\"https:\\\/\\\/flcube.com\"],\"url\":\"https:\\\/\\\/flcube.com\\\/?author=1\"}]}<\/script>\n<!-- \/ Yoast SEO Premium plugin. -->","yoast_head_json":{"title":"HuidaGene Receives CDE IND Approval for Ophthalmology Gene Therapy HG004 - Insight, China&#039;s Pharmaceutical Industry","description":"China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced receiving Investigational New Drug (IND) approval from the Center for Drug Evaluation (CDE) in China for its ophthalmology gene therapy, HG004. 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