Passage Bio Announces Positive Feedback from FDA on Expansion of upliFT-D Trial of PBFT02 to Include FTD-C9orf72 Patients

Core Insights - Passage Bio has received positive feedback from the FDA regarding its proposal to evaluate PBFT02 for treating frontotemporal dementia (FTD) patients with C9orf72 gene mutations, indicating alignment on the trial expansion [12] - The company plans to initiate dosing of FTD-C9orf72 patients in the first half of 2025, following the amendment of the ongoing upliFT-D Phase 1/2 global study protocol [13] Company Overview - Passage Bio is a clinical-stage genetic medicines company focused on developing therapies for neurodegenerative diseases, with PBFT02 as its lead product candidate aimed at elevating progranulin (PGRN) levels to restore lysosomal function and slow disease progression [15] - PBFT02 utilizes an AAV1 viral vector for delivering a functional GRN gene through ICM administration, targeting the underlying pathology of neurodegenerative conditions [14] Clinical Development - The FDA has granted PBFT02 Fast Track and Orphan Drug designations, which may facilitate its development and approval process [7] - Preclinical studies have shown that increasing PGRN levels can reduce TDP-43 pathology and slow neurodegeneration, supporting the potential efficacy of PBFT02 [8][12]