Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, gives an overview of frontotemporal dementia (FTD) with granulin (GRN) mutations.
As Dr. Forman explains, 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 involves mutations in the GRN gene. These mutations cause a deficiency of progranulin, which plays important roles in cell biology, development, and inflammation.
Recently, Passage Bio announced they have dosed the first patient at the first patient in the global phase 1/2 upliFT-D clinical trial that is evaluating PBFT02, an adeno-associated virus (AAV) delivery gene therapy for the treatment of patients with FTD and GRN mutations.
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