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 has no approved disease-modifying therapies. In approximately 5–10% 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.
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:
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.
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