{"id":235633,"date":"2026-04-20T23:02:41","date_gmt":"2026-04-21T04:02:41","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2026\/04\/uniqures-path-for-huntingtons-gene-therapy-clouded-by-ethical-questions-as-potential-phase-3-looms"},"modified":"2026-04-20T23:02:41","modified_gmt":"2026-04-21T04:02:41","slug":"uniqures-path-for-huntingtons-gene-therapy-clouded-by-ethical-questions-as-potential-phase-3-looms","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2026\/04\/uniqures-path-for-huntingtons-gene-therapy-clouded-by-ethical-questions-as-potential-phase-3-looms","title":{"rendered":"UniQure\u2019s Path for Huntington\u2019s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/uniqures-path-for-huntingtons-gene-therapy-clouded-by-ethical-questions-as-potential-phase-3-looms.jpg\"><\/a><\/p>\n<p>UniQure\u2019s highly promising Huntington\u2019s disease gene therapy BLA (biologics license application) was rejected by the FDA \u2014 because UniQure used an external control rather than a surgical sham control. Yet the latter would put control group patients at additional risk, making it ethically problematic. Hopefully some agreement will be reached which circumvents these issues! For now, it is an educational story to watch unfold.<\/p>\n<hr>\n<p>Abi-Saab said during uniQure\u2019s earnings call that he wouldn\u2019t count on the four-year data altering the FDA\u2019s decision.<\/p>\n<p>\u201cWe don\u2019t believe that there\u2019s any reason we have today to believe that this will change the FDA\u2019s position regarding the Phase 1\/2 trials,\u201d he told investors.<\/p>\n<p>H.C. Wainwright struck a different tone, however, in the Monday note. \u201cWhile the FDA appears to be enforcing a full sham surgery-controlled Phase 3 trial in Huntington\u2019s, we believe an alternative path forward may be negotiated given the strong AMT-130 data generated to-date,\u201d the analysts wrote. The 4-year data \u201cshould further inform the durability and magnitude of effect observed to date.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"<p>UniQure\u2019s highly promising Huntington\u2019s disease gene therapy BLA (biologics license application) was rejected by the FDA \u2014 because UniQure used an external control rather than a surgical sham control. Yet the latter would put control group patients at additional risk, making it ethically problematic. Hopefully some agreement will be reached which circumvents these issues! For [\u2026]<\/p>\n","protected":false},"author":636,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,32],"tags":[],"class_list":["post-235633","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-education"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/235633","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/636"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=235633"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/235633\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=235633"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=235633"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=235633"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}