A Study of PBFT02 in Patients With Frontotemporal Dementia and Progranulin Mutations (FTD-GRN)

PBFT02 is an adeno-associated viral vector serotype 1 carrying GRN, the gene encoding for human progranulin, formulated as a solution for injection into the cisterna magna. This is a global interventional, multicenter, open-label, single-arm, dose-escalation study of PBFT02 delivered as a one-time dose administered into the cisterna magna to patients with FTD-GRN. Subjects aged ≥ 35 and ≤ 75 years with early symptomatic FTD-GRN may be enrolled into the study. Two dose levels of PBFT02 will be studied in patients with FTD-GRN. The study will sequentially enroll 2 cohorts. An optional third dose level cohort may be enrolled based on the results of the first two cohorts. This is a 5-year study, with a 2-year main study, followed by a 3-year safety extension.