Passage Bio Inc. has released a corporate presentation detailing advancements in its clinical and preclinical programs targeting neurodegenerative diseases. The company is progressing a clinical-stage, one-time gene therapy, PBFT02, for frontotemporal dementia caused by progranulin gene (_GRN_) mutations (FTD-GRN). PBFT02 utilizes a proprietary AAV1 vector and is administered via a nonsurgical injection directly to the cerebrospinal fluid, aiming to restore progranulin protein levels. Interim data indicate durable and elevated CSF progranulin levels following treatment. PBFT02 has received Fast Track and Orphan Drug Designation. Additionally, Passage Bio is pursuing preclinical development of a differentiated gene therapy for Huntington’s disease. The company estimates its cash runway extends into the first quarter of 2027. You can access the full presentation through the link below.

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