PBFT02 delivers superior CSF progranulin with AAV1 vector; interim safety and biomarker data promising

Passage BIO, Inc.

Capsid comparison in NHPs: AAV1 vector achieved superior human PGRN levels in CSF versus AAV5 and AAVhu68 after ICM administration.
In Grn knockout mice, PBFT02 improved lysosomal histopathology and reduced neuroinflammation across brain regions.
Interim clinical data from upliFT-D trial: Dose 1 well-tolerated; durable CSF progranulin increases sustained up to 12 months.
NHP biodistribution showed ICM administration of PBFT02 achieved high gene distribution to cortical and sub-cortical brain regions and spinal cord.

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