PASG - Passage Bio to start testing PBFT02 gene therapy in patients with a type of dementia

The FDA has signed off investigational new drug application for Passage Bio's ([[PASG]] -0.9%) PBFT02, an adeno-associated virus-delivery gene therapy for the treatment of patients with Frontotemporal Dementia ((FTD)), a form of early onset dementia, with granulin mutations.The company expects to initiate a Phase 1/2 trial for PBFT02 in 1H of 2021, designed as a dose-escalation study with primary endpoint as safety and tolerability; secondary endpoints include CSF progranulin levels, disease biomarkers, and clinical outcome measure.Initial data is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.The FDA has granted an Orphan Drug designation for PBFT02 for the treatment of FTD-GRN.

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Passage Bio to start testing PBFT02 gene therapy in patients with a type of dementia