{"id":326382,"date":"2026-08-21T03:07:04","date_gmt":"2026-08-21T03:07:04","guid":{"rendered":"https:\/\/www.newsbeep.com\/us-ny\/326382\/"},"modified":"2026-08-21T03:07:04","modified_gmt":"2026-08-21T03:07:04","slug":"andelyn-partners-with-queens-university-on-aav9-gm2-therapy","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us-ny\/326382\/","title":{"rendered":"Andelyn partners with Queen’s University on AAV9-GM2 therapy"},"content":{"rendered":"

Cell and gene therapy (CGT) focused contract development and manufacturing organization (CDMO) Andelyn Biosciences announced a partnership<\/a> with Queen\u2019s University at Kingston to advance AAV9-GM2. The gene therapy candidate is under development to treat GM2 gangliosidoses, which Matt Niloff, CCO of Andelyn Biosciences, described to BioProcess International as \u201ca group of rare, inherited disorders that can cause profound neurological decline.\u201d<\/p>\n

Under the agreement, Andelyn will use its AAV Curator platform to advance the AAV9-GM2 program. According to the company, the AAV Curator platform offers a modular approach with configurable materials that can adapt to a given program\u2019s requirements.<\/p>\n

\u201cAndelyn\u2019s AAV experience began in 1992 through gene therapy work at Nationwide Children\u2019s Hospital, with cGMP production of the first patient doses starting in 2006,\u201d Niloff told us. The company developed the AAV Curator platform in 2009, which has since evolved through the firm\u2019s experience in development, clinical manufacturing, and scale-up. \u201cThat history matters for the AAV9-GM2 program because the platform is not a one-size-fits-all process.\u201d<\/p>\n

Related:Bristol Myers Squibb picks Houston for multi-modality $2.3bn manufacturing campus<\/a><\/p>\n

Andelyn\u2019s facility in Dublin, Ohio, will support early process and development activities with clinical GMP manufacturing taking place at the firm\u2019s Columbus site. Niloff emphasized the importance of having two sites that work well together. \u201cThat close connection is especially important for a program such as AAV9-GM2, where maintaining process understanding, product quality, and continuity as the program advances toward patient dosing is critical.\u201d<\/p>\n

According to a report<\/a> by The Kingston Whig Standard, the Hilary and Galen Weston Foundation awarded Queen\u2019s University C$5 million (US$3.56 million) toward its research into GM2 gangliosidoses in May 2026. Jagdeep Walia, medical geneticist and professor in the Department of Pediatrics at Queen’s University, said the money will help launch clinical trials.<\/p>\n

Upon announcement of the partnership with Andelyn, Walia praised the CDMO for its body of work. “This partnership with Andelyn is very strategic as they have the established expertise in producing vectors for many gene therapy programs. Their track record and ability to manufacture the viral vector for the upcoming GM2 program gives us confidence that the participants in the clinical trial will be receiving the highest quality, safe product.”<\/p>\n

Niloff told us that Andelyn is proud to support Queen\u2019s University in its effort. \u201cThe work reflects the focused scientific effort essential to rare disease: moving a promising therapeutic approach toward a clinical trial where it may ultimately make a meaningful difference for patients and families.\u201d<\/p>\n

Related:Video: Lonza executive discusses the evolving CGT landscape<\/a><\/p>\n

\u201cThe patient is the top priority,\u201d he said. \u201cPeople and families affected by GM2 gangliosidoses are waiting for new treatment options, so there is genuine urgency to move the program forward.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Cell and gene therapy (CGT) focused contract development and manufacturing organization (CDMO) Andelyn Biosciences announced a partnership with…\n","protected":false},"author":2,"featured_media":326383,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[37],"tags":[9,24,63,122,124,123],"class_list":["post-326382","post","type-post","status-publish","format-standard","has-post-thumbnail","category-queens","tag-new-york","tag-new-york-city","tag-nyc","tag-queens","tag-queens-headlines","tag-queens-news"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts\/326382","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/comments?post=326382"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts\/326382\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/media\/326383"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/media?parent=326382"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/categories?post=326382"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/tags?post=326382"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}