Passage Bio is a clinical-stage genetic medicines company developing one-time gene therapies for neurodegenerative diseases using adeno-associated virus (AAV) vectors to deliver functional genes directly to the brain and spinal cord. The company's lead candidate, PBFT02, is a gene replacement therapy targeting frontotemporal dementia caused by progranulin deficiency and other CNS indications including another form of frontotemporal dementia, amyotrophic lateral sclerosis, and Alzheimer's disease, with regulatory orphan designations and fast-track status. Passage Bio generates no current revenue as a clinical-stage company and plans to pursue regulatory feedback and conduct registrational trials, while maintaining partnerships with contract manufacturers and retaining options to expand its pipeline through collaborations targeting additional neurodegenerative conditions.