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# Passage Bio Cleared for Gene Therapy Trial
- URL: https://www.fdaweb.com/passage-bio-cleared-for-gene-therapy-trial/
- Published: 2021-01-28T12:00:00.000Z
- Updated: 2026-09-14T16:44:35.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148552

FDA has cleared a Passage Bio IND application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for treating patients with frontotemporal dementia (FTD) with granulin (GRN) mutations. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies, the company says.

Passage Bio expects to begin a Phase1/2 clinical trial for PBFT02 in the first half of this year. The trial is designed as a dose-escalation study of a single dose of PBFT02 in subjects with FTD and heterozygous mutations in the GRN gene. The primary endpoint is safety and tolerability, while secondary endpoints include progranulin levels, disease biomarkers, and clinical outcome measures.

Passage Bio says it is developing PBFT02 as a single dose delivered via intra-cisterna magna injection. The gene therapy uses an AAV1 viral vector to deliver a modified DNA that encodes the GRN gene to a patient’s cells. The goal of this vector and delivery approach is to provide higher than normal progranulin (PGRN) levels to the central nervous system to overcome the PGRN deficiency in GRN mutation carriers, it says.

Passage Bio is advancing therapy with preclinical data generated by its collaborator, University of Pennsylvania's Gene Therapy Program. “The data, published in the peer-reviewed scientific journal *Annals of Clinical and Translational Neurology*, showed that a single administration of an optimized AAV containing the GRN gene resulted in elevated levels of PGRN in the brain and cerebral spinal fluid, reduced lysosomal storage lesions, normalized lysosomal enzyme expression and corrected microgliosis in a mouse model of progranulin deficiency, the company says.