Passage Bio to present data from Cohort 1 patients evaluating PBFT02

Passage Bio will present updated data from the ongoing global Phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus-delivery gene therapy for the treatment of patients with frontotemporal dementia with granulin mutations, at the 14th International Conference on Frontotemporal Dementias. The poster presentation will include safety and biomarker data from all treated patients in the first cohort of the study. Dose 1 of PBFT02 was well-tolerated in all patients who received an enhanced immunosuppression regimen following protocol amendment. Dose 1 of PBFT02 treatment resulted in a robust and durable increase in PGRN expression. Relative to baseline, PBFT02 increased CSF PGRN expression in all patients; up to 6-fold at one month and up to 10-fold at six months. The effect of PBFT02 was consistent across all patients, with CSF PGRN levels exceeding the range found in healthy adult controls of 3.3 to 8.2 ng/mL.