PBFT02

PBFT02 is an investigational gene therapy designed to treat neurodegenerative diseases, primarily frontotemporal dementia (FTD) associated with mutations in the GRN gene (FTD-GRN) and C9orf72 gene (FTD-C9orf72). It utilizes a non-replicating recombinant adeno-associated virus serotype 1 (AAV1) vector to deliver a functional copy of the human GRN gene encoding progranulin directly into the central nervous system via intra-cisterna magna administration. The goal is to elevate progranulin levels in the brain and cerebrospinal fluid, thereby restoring lysosomal function, reducing neuroinflammation, and slowing disease progression. Preclinical studies have shown broad CNS distribution of the vector and robust increases in CSF progranulin levels. PBFT02 has received Fast Track and Orphan Drug designations from regulatory agencies[1][2][3][4][5][6].