• Passage Bio highlighted PBFT02 as a one-time AAV1 gene therapy for FTD caused by GRN mutations, delivered via a non-surgical intracisterna magna injection to cerebrospinal fluid.
• Interim clinical data showed durable increases in CSF progranulin, including mean CSF progranulin of 22.8 ng/mL at 12 months in Dose 1 patients (n=6), with durability reported out to 18 months (n=3).
• Biomarker comparisons versus ALLFTD natural history in baseline CDR 1 patients showed a 64% reduction in whole brain atrophy at 12 months (-3.1% for PBFT02, n=2, versus -8.7% for natural history, n=7), with a 54% reduction in frontotemporal cortex atrophy (-4.6% versus -9.9%).
• Safety snapshot as of March 23, 2026 covered 11 patients; PBFT02 was generally well tolerated, with 2 patients reporting serious treatment-related adverse events, all asymptomatic, including venous sinus thrombosis (2) and an LFT increase (1).
• Cash resources totaled $33.3 million as of March 31, 2026, with a Huntington’s disease gene therapy program described as preclinical.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Passage Bio Inc. published the original content used to generate this news brief on May 12, 2026, and is solely responsible for the information contained therein.