Passage Bio Announces Initial Clinical Data From Phase 1/2 UpliFT-D Trial On PBFT02

(RTTNews) - Clinical-stage genetic medicines company Passage Bio, Inc. (PASG) Wednesday announced initial clinical data from three Cohort 1 patients in the ongoing global Phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus-delivery gene therapy.

This treatment is intended for patients with frontotemporal dementia with granulin mutations. frontotemporal dementia is an early onset dementia, due to mutations in the GRN gene, causing a deficiency of progranulin or PGRN. The company noted that the emerging evidence suggests that PGRN deficiency may contribute to lysosomal dysfunction. It has no approved disease-modifying therapies.

Mark Forman, chief medical officer at Passage Bio said, the upliFT-D clinical trial showcased the ability of PBFT02 to elevate CSF progranulin to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment. "We believe these data, surpassing our expectations based on preclinical non-human primate models, validate the compelling potential of PBFT02 to address progranulin deficiency—a key driver of disease progression in individuals with FTD-GRN."