Biopharmaceutical company Passage Bio has highlighted significant progress in its phase 1/2 clinical trial, known as upliFT-D, targeting a genetic form of Frontotemporal Dementia (FTD). The trial evaluates PBFT02, an experimental gene therapy designed to treat FTD caused by mutations in the GRN gene, which leads to a deficiency in the protein progranulin. PBFT02 utilizes an engineered virus to deliver a healthy copy of the GRN gene.
In late 2023, Passage Bio announced promising preliminary results from the trial, with updated interim data showing continued elevation in cerebrospinal fluid progranulin levels six months post-treatment in two patients. This consistent response to PBFT02 underscores the therapy's potential impact at the current dose. Passage Bio has completed dosing for all five patients in Cohort 1 and plans to begin dosing a second cohort in the first half of 2024.
Additional safety and biomarker data from the first cohort are anticipated in the latter half of 2024, with 12-month follow-up data expected in the first half of 2025. The company is also evaluating multiple individuals with GRN mutations for study eligibility across trial sites in Brazil, Canada, the United States, and Europe.
Furthermore, Passage Bio has initiated a process with the U.S. Food and Drug Administration to receive feedback on its plans to treat FTD caused by C9orf72 genetic mutations. Similar to FTD-GRN, FTD-C9orf72 is associated with abnormal accumulations of the protein TDP-43. Research indicates that elevating progranulin levels can mitigate TDP-43-related pathology and slow neurodegeneration. The company expects FDA feedback later this year.
Approximately 40% of individuals diagnosed with FTD have a family history of the condition, known as familial FTD, with a subset of these cases caused by known genetic mutations such as GRN or C9orf72.
