Passage Bio delivered preclinical, interim data at ESGCT

Passage Bio (PASG) announced the company delivered preclinical and interim clinical data as part of an oral presentation at the European Society of Gene & Cell Therapy, ESGCT, 31st Annual Congress being held October 22-25, 2024, in Rome, Italy. Title: “Non-clinical and early clinical development of PBFT02, an AAV gene therapy for frontotemporal dementia with GRN mutations.” Data Summary: Capsid comparison study in non-human primates showed the AAV1 vector achieved superior human PGRN levels in the CSF as compared to AAV5 and AAVhu6 following intra-cisterna magna administration; Dose escalation study in Grn knockout mice showed PBFT02 improved lysosomal histopathology and reduced neuroinflammation throughout the brain following intra-CSF delivery; NHP biodistribution study showed ICM administration of PBFT02 achieved high levels of gene distribution throughout the nervous system; PBFT02 was well tolerated in NHPs, and ICM administration resulted in dose-dependent PGRN elevations in NHP CSF; Interim safety and biomarker data from the upliFT-D clinical study demonstrated that Dose 1 of PBFT02 was generally well-tolerated after ICM administration and led to consistent, durable increases in levels of CSF progranulin in all treated Cohort 1 patients