Passage Bio is a clinical-stage biotechnology company specializing in the development of gene therapies for rare monogenic diseases of the central nervous system. Leveraging a proprietary adeno-associated virus (AAV) platform, the company focuses on delivering functional copies of disease-causing genes directly to affected tissues. Its research activities center on addressing unmet medical needs in genetic disorders where conventional treatments are limited or nonexistent.
The company’s lead programs target GM1 gangliosidosis and Tay-Sachs disease, both fatal neurodegenerative conditions arising from enzyme deficiencies. In parallel, Passage Bio is advancing preclinical candidates for GM2 gangliosidosis, Krabbe disease and Alexander disease. By collaborating with academic institutions and patient advocacy groups, the company conducts natural history studies, refines dosing strategies and accelerates regulatory pathways to facilitate clinical development.
Founded in 2019 as a spin-out from the Gene Therapy Program at Children’s Hospital of Philadelphia and the University of Pennsylvania, Passage Bio has established its headquarters in Philadelphia, Pennsylvania. The company operates a research facility in the greater Philadelphia area and engages with global experts in gene therapy from leading academic medical centers. Through partnerships and grant collaborations, Passage Bio extends its reach to international research communities and patient networks.
Under the leadership of President and Chief Executive Officer Paul R. Miller, Passage Bio’s executive team combines expertise in neurology, molecular biology and regulatory affairs. The company’s board of directors includes seasoned professionals from both biotech and pharmaceutical industries, guiding strategic decisions and supporting the advancement of its gene therapy portfolio. Passage Bio remains committed to pioneering durable treatments for patients facing devastating genetic disorders.
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