{"id":23181,"date":"2023-01-04T16:12:00","date_gmt":"2023-01-04T08:12:00","guid":{"rendered":"https:\/\/flcube.com\/?p=23181"},"modified":"2025-01-26T16:39:29","modified_gmt":"2025-01-26T08:39:29","slug":"canbridge-secures-global-rights-to-second-generation-gene-therapy-for-sma","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=23181","title":{"rendered":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA"},"content":{"rendered":"\n

China – based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228<\/a>) has unveiled a license deal to secure exclusive global rights to a novel, second – generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 – SMN1p – co – hSMN1, a self – complementary AAV9 gene therapy expressing a codon – optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.<\/p>\n\n\n\n

About SMA and the Gene Therapy<\/strong>
Spinal muscular atrophy (SMA) is a rare genetic disease caused by the lack of a functional survival motor neuron 1 (SMN1) gene, which codes for a protein essential to motor neuron survival. The loss of motor neurons leads to progressive muscle weakness and death at an early age. The most common form of the disease appears between birth and 6 months, often leading to death by the age of 2 if untreated. Gene therapies, such as the one licensed by CANbridge, are designed to deliver a fully functional human SMN1 gene via a viral vector that enters the spinal cord, halting the degeneration of motor neurons.<\/p>\n\n\n\n

Development and Preclinical Data<\/strong>
CANbridge has been collaborating with UMass Chan Medical School on the development of this gene therapy, sponsoring preclinical development to assess its potential to outperform the currently approved first – generation gene therapy, Novartis’s Zolgensma (onasemnogene abeparvovec). Animal data presented at various meetings in 2022, including the American Society of Gene and Cell Therapy (ASGCT), showed that UMass’s candidate therapy outperformed a benchmark similar to Zolgensma in several key endpoints, including reduced liver toxicity. The key difference lies in the choice of vector, with UMass’s second – generation vector utilizing the endogenous SMN1 promoter to control gene expression in different tissues.<\/p>\n\n\n\n

Expansion of Gene Therapy Portfolio<\/strong>
CANbridge is building a specialist gene therapy portfolio and also announced an amendment to an earlier April 2021 deal with US – headquartered LogicBio Therapeutics Inc. (LogicBio, NASDQ: LOGC). The original deal granted exclusive global rights to develop, manufacture, and commercialize LogicBio’s adeno – associated virus (AAV) sL65 for Fabry and Pompe gene therapies. Under this week’s amendment, CANbridge is also granted non – exclusive global rights to LogicBio’s proprietary manufacturing process for Fabry\/Pompe gene therapies. Option rights to sL65 – based therapies for two additional indications and to a second therapy, LB – 001, have been removed from the agreement.-Fineline Info & Tech<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

China – based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to…<\/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":[7,10],"tags":[142,66,883,3313,140],"class_list":["post-23181","post","type-post","status-publish","format-standard","hentry","category-company","category-deals","tag-canbridge-pharmaceuticals","tag-gene-therapy","tag-hkg-1228","tag-logicbio-therapeutics","tag-novartis"],"yoast_head":"\nCANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA - Insight, China's Pharmaceutical Industry<\/title>\n<meta name=\"description\" content=\"China - based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to secure exclusive global rights to a novel, second - generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 - SMN1p - co - hSMN1, a self - complementary AAV9 gene therapy expressing a codon - optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.\" \/>\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=23181\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA\" \/>\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. Stay informed, stay ahead with FLCUBE.COM\" \/>\n<meta property=\"og:url\" content=\"https:\/\/flcube.com\/?p=23181\" \/>\n<meta property=\"og:site_name\" content=\"Insight, China's Pharmaceutical Industry\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/profile.php?id=61566606313834\" \/>\n<meta property=\"article:published_time\" content=\"2023-01-04T08:12:00+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-01-26T08:39:29+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/flcube.com\/wp-content\/uploads\/2024\/11\/Fineline-info-tech-scaled.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"2560\" \/>\n\t<meta property=\"og:image:height\" content=\"1894\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Fineline Cube\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Fineline Cube\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181\"},\"author\":{\"name\":\"Fineline Cube\",\"@id\":\"https:\\\/\\\/flcube.com\\\/#\\\/schema\\\/person\\\/19ad11870d326204db8524d3c3c5e66a\"},\"headline\":\"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA\",\"datePublished\":\"2023-01-04T08:12:00+00:00\",\"dateModified\":\"2025-01-26T08:39:29+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181\"},\"wordCount\":426,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/#organization\"},\"keywords\":[\"CANbridge Pharmaceuticals\",\"Gene therapy\",\"HKG: 1228\",\"LogicBio Therapeutics\",\"Novartis\"],\"articleSection\":[\"Company\",\"Deals\"],\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\\\/\\\/flcube.com\\\/?p=23181#respond\"]}],\"copyrightYear\":\"2023\",\"copyrightHolder\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/#organization\"}},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181\",\"url\":\"https:\\\/\\\/flcube.com\\\/?p=23181\",\"name\":\"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA - Insight, China's Pharmaceutical Industry\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/#website\"},\"datePublished\":\"2023-01-04T08:12:00+00:00\",\"dateModified\":\"2025-01-26T08:39:29+00:00\",\"description\":\"China - based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to secure exclusive global rights to a novel, second - generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 - SMN1p - co - hSMN1, a self - complementary AAV9 gene therapy expressing a codon - optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/flcube.com\\\/?p=23181\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/flcube.com\\\/?p=23181#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/flcube.com\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA\"}]},{\"@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":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA - Insight, China's Pharmaceutical Industry","description":"China - based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to secure exclusive global rights to a novel, second - generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 - SMN1p - co - hSMN1, a self - complementary AAV9 gene therapy expressing a codon - optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/flcube.com\/?p=23181","og_locale":"en_US","og_type":"article","og_title":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA","og_description":"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. Stay informed, stay ahead with FLCUBE.COM","og_url":"https:\/\/flcube.com\/?p=23181","og_site_name":"Insight, China's Pharmaceutical Industry","article_publisher":"https:\/\/www.facebook.com\/profile.php?id=61566606313834","article_published_time":"2023-01-04T08:12:00+00:00","article_modified_time":"2025-01-26T08:39:29+00:00","og_image":[{"width":2560,"height":1894,"url":"https:\/\/flcube.com\/wp-content\/uploads\/2024\/11\/Fineline-info-tech-scaled.jpg","type":"image\/jpeg"}],"author":"Fineline Cube","twitter_card":"summary_large_image","twitter_misc":{"Written by":"Fineline Cube","Est. reading time":"2 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/flcube.com\/?p=23181#article","isPartOf":{"@id":"https:\/\/flcube.com\/?p=23181"},"author":{"name":"Fineline Cube","@id":"https:\/\/flcube.com\/#\/schema\/person\/19ad11870d326204db8524d3c3c5e66a"},"headline":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA","datePublished":"2023-01-04T08:12:00+00:00","dateModified":"2025-01-26T08:39:29+00:00","mainEntityOfPage":{"@id":"https:\/\/flcube.com\/?p=23181"},"wordCount":426,"commentCount":0,"publisher":{"@id":"https:\/\/flcube.com\/#organization"},"keywords":["CANbridge Pharmaceuticals","Gene therapy","HKG: 1228","LogicBio Therapeutics","Novartis"],"articleSection":["Company","Deals"],"inLanguage":"en-US","potentialAction":[{"@type":"CommentAction","name":"Comment","target":["https:\/\/flcube.com\/?p=23181#respond"]}],"copyrightYear":"2023","copyrightHolder":{"@id":"https:\/\/flcube.com\/#organization"}},{"@type":"WebPage","@id":"https:\/\/flcube.com\/?p=23181","url":"https:\/\/flcube.com\/?p=23181","name":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA - Insight, China's Pharmaceutical Industry","isPartOf":{"@id":"https:\/\/flcube.com\/#website"},"datePublished":"2023-01-04T08:12:00+00:00","dateModified":"2025-01-26T08:39:29+00:00","description":"China - based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to secure exclusive global rights to a novel, second - generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 - SMN1p - co - hSMN1, a self - complementary AAV9 gene therapy expressing a codon - optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.","breadcrumb":{"@id":"https:\/\/flcube.com\/?p=23181#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/flcube.com\/?p=23181"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/flcube.com\/?p=23181#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/flcube.com\/"},{"@type":"ListItem","position":2,"name":"CANbridge Secures Global Rights to Second-Generation Gene Therapy for SMA"}]},{"@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"}]}},"jetpack_publicize_connections":[],"jetpack_featured_media_url":"","jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/posts\/23181","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/flcube.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=23181"}],"version-history":[{"count":1,"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/posts\/23181\/revisions"}],"predecessor-version":[{"id":23184,"href":"https:\/\/flcube.com\/index.php?rest_route=\/wp\/v2\/posts\/23181\/revisions\/23184"}],"wp:attachment":[{"href":"https:\/\/flcube.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=23181"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/flcube.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=23181"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/flcube.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=23181"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}