{"id":159286,"date":"2023-03-01T13:22:23","date_gmt":"2023-03-01T19:22:23","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2023\/03\/gene-therapy-clinical-trial-for-frontotemporal-dementia-has-begun"},"modified":"2023-03-01T13:22:23","modified_gmt":"2023-03-01T19:22:23","slug":"gene-therapy-clinical-trial-for-frontotemporal-dementia-has-begun","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2023\/03\/gene-therapy-clinical-trial-for-frontotemporal-dementia-has-begun","title":{"rendered":"Gene Therapy Clinical Trial for Frontotemporal Dementia Has Begun"},"content":{"rendered":"<p><\/p>\n<p><iframe style=\"display: block; margin: 0 auto; width: 100%; aspect-ratio: 4\/3; object-fit: contain;\" src=\"https:\/\/www.youtube.com\/embed\/F2IY92Hrdqg?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; encrypted-media; gyroscope;\n   picture-in-picture\" allowfullscreen><\/iframe><\/p>\n<p>Bruce Willis has FTD. I always wondered if gene therapy could help. Apparently so did Passage Bio, and they are doing clinical trials.<\/p>\n<p>FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control personality, executive function, and language. FTD is a form of early onset dementia and currently has no approved disease-modifying therapies. In approximately 5\u201310% of individuals with FTD, the disease occurs because of mutations in the GRN gene. These mutations cause a deficiency of progranulin that helps regulate cellular processes.<\/p>\n<p>Recently, Passage Bio announced that the first patient has been dosed in the global phase 1\/2 upliFT-D clinical trial evaluating PBFT02. As Dr. Forman explains, PBFT02 is an AAV delivery gene therapy for the treatment of patients with FTD with GRN mutations. The upliFT-D trial is a dose-escalation study in which two doses will be sequentially evaluated in two cohorts, with a possible third cohort. Inclusion criteria for the trial include that patients:<\/p>\n<hr>\n<p>Dr. Mark Forman, Chief Medical Officer at Passage Bio, discusses the phase 1\/2 upliFT-D clinical trial evaluating PBFT02, an AAV delivery gene therapy for patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Bruce Willis has FTD. I always wondered if gene therapy could help. Apparently so did Passage Bio, and they are doing clinical trials. FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control personality, executive function, and language. FTD is a form of early onset dementia and currently [\u2026]<\/p>\n","protected":false},"author":534,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,47],"tags":[],"class_list":["post-159286","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-neuroscience"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/159286","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\/534"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=159286"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/159286\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=159286"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=159286"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=159286"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}