This study tests a one-time gene therapy called PBFT02 for frontotemporal dementia (FTD) in adults who have a mutation in the GRN or C9ORF72 gene. The therapy aims to deliver a working copy of the GRN gene to the brain to help control the disease. About 30 participants will recei…
Phase: PHASE1, PHASE2 • Sponsor: Passage Bio, Inc. • Aim: Disease control
Last updated May 17, 2026 04:29 UTC