PBGM01
Infantile GM1 Gangliosidosis
Phase 1/2Active
Key Facts
About Passage Bio
Passage Bio is a clinical-stage biotech focused on developing AAV-based gene therapies for rare, monogenic CNS disorders. The company's mission is to deliver one-time, transformative treatments that permanently redefine the course of neurodegenerative diseases. Its strategy centers on two lead Phase 1/2 programs—PBFT02 for FTD-GRN and PBGM01 for GM1 gangliosidosis—supported by a technology platform emphasizing optimized delivery and manufacturing. Despite significant clinical and financial challenges common to the gene therapy sector, Passage Bio represents a focused, platform-driven approach to treating devastating neurological conditions.
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