Develops gene therapies for rare monogenic central nervous system disorders, focusing on delivering transformative treatments.
Passage Bio, Inc., headquartered in Philadelphia, Pennsylvania, is a pioneering genetic medicines company focused on developing groundbreaking therapies for diseases of the central nervous system. Established in 2017, Passage Bio is dedicated to advancing transformative treatments using innovative genetic approaches. The company's pipeline includes PBGM01, PBFT02, PBKR03, PBML04, PBAL05, and PBCM06, each targeting specific genetic disorders such as infantile GM1, FTD-GRN, infantile Krabbe disease, metachromatic leukodystrophy, amyotrophic lateral sclerosis (ALS), and Charcot-Marie-Tooth Type 2A, respectively.
Key to Passage Bio's therapeutic innovations is its utilization of proprietary AAV capsids, including AAVhu68 and AAV1, designed to effectively deliver functional genes to both the brain and peripheral tissues. This approach aims to address the underlying genetic causes of debilitating CNS diseases, offering hope to patients and their families. The company's strategic collaborations, including partnerships with the Trustees of the University of Pennsylvania's Gene Therapy Program and Catalent Maryland, Inc., underscore its commitment to accelerating the development and delivery of novel genetic therapies.
With a robust research and development strategy, Passage Bio continues to expand its capabilities in genetic medicine, leveraging cutting-edge technology and scientific expertise. By focusing on rare and severe neurological conditions, Passage Bio strives to make a meaningful impact on patient outcomes, pushing the boundaries of genetic medicine to transform the treatment landscape for CNS disorders worldwide.