{"id":184726,"date":"2025-09-27T06:36:09","date_gmt":"2025-09-27T06:36:09","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/184726\/"},"modified":"2025-09-27T06:36:09","modified_gmt":"2025-09-27T06:36:09","slug":"cgtlives-weekly-rewind-september-26-2025","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/184726\/","title":{"rendered":"CGTLive\u00ae\u2019s Weekly Rewind \u2013 September 26, 2025"},"content":{"rendered":"\n

Welcome to CGTLive\u00ae\u2019s Weekly Rewind! We\u2019ve compiled 5 highlights from this week\u2019s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.<\/p>\n

1. Phase 1\/2 Trial for uniQure’s Huntington Disease Gene Therapy AMT-130 Hits Primary End Point<\/a><\/p>\n

The study also met a key secondary end point.<\/p>\n

2. Jainu Jogani on the Potential to Address CDKL5 With Gene Therapy<\/a><\/p>\n

The cofounder of Child\u2019s Cure Genetic Research discussed his daughter\u2019s rare genetic disease and the need for new treatment options.<\/p>\n

3. Atsena Therapeutics\u2019 Trial for X-Linked Retinoschisis Gene Therapy ATSN-201 Finishes Dosing Adult Patients in Part B<\/a><\/p>\n

Part B of LIGHTHOUSE enrolled 9 patients and is expected to enroll 3 pediatric patients.<\/p>\n

4. The Importance of Addressing Gene Therapy’s Financial Viability in Ultra Rare Diseases<\/a><\/p>\n

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed a trend of doctors and scientists working on models to make gene therapy products commercially viable.<\/p>\n

5. Capricor Therapeutics Testing DMD Cell Therapy Deramiocel\u2019s Mettle in Phase 3 HOPE-3 Clinical Trial<\/a><\/p>\n

In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive\u00ae is taking a closer look at this ongoing study.<\/p>\n","protected":false},"excerpt":{"rendered":"Welcome to CGTLive\u00ae\u2019s Weekly Rewind! We\u2019ve compiled 5 highlights from this week\u2019s coverage of advances in gene and…\n","protected":false},"author":2,"featured_media":184727,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[50],"tags":[200,79],"class_list":["post-184726","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","tag-genetics","tag-science"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/184726","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=184726"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/184726\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/184727"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=184726"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=184726"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=184726"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}