Passage Bio, Inc. delivered preclinical and interim clinical data for PBFT02 in Frontotemporal Dementia with granulin (FTD-GRN) at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress on October 24, 2024.
Preclinical data demonstrated that an AAV1 vector achieved superior human progranulin levels in the cerebrospinal fluid (CSF) compared to AAV5 and AAVhu68. Nonclinical data also showed PBFT02 improved lysosomal histopathology, reduced neuroinflammation in Grn knockout mice, and achieved widespread vector distribution throughout the nervous system in non-human primates.
Interim clinical data from the upliFT-D trial highlighted a well-tolerated safety profile and consistent, durable increases in CSF progranulin levels. These findings validate the preclinical results and position PBFT02 as a potential progranulin-raising therapy for FTD-GRN.
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