Passage Bio Licenses Three Pediatric Gene Therapies to GEMMA and Starts New Research Collaboration

Passage Bio, Inc., a clinical-stage company specializing in genetic medicines for neurodegenerative diseases, announced a significant partnership with GEMMA Biotherapeutics, Inc. (GEMMABio). This newly established firm, co-founded by Dr. James M. Wilson, will take charge of Passage Bio’s three clinical-stage pediatric lysosomal storage disease programs. These programs include PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy, with GEMMABio granted exclusive global rights for their development and commercialization.

The licensing deal will provide Passage Bio with an initial payment of $10 million for clinical product supply, with an additional $10 million contingent on achieving certain business milestones by GEMMABio. Furthermore, Passage Bio stands to receive up to $114 million in development and commercial milestones, along with future royalties. In return, GEMMABio will gain sublicenses for relevant intellectual property and handle all clinical trial materials and product supply obligations for the licensed programs. Additionally, GEMMABio will assume remaining financial duties owed to the University of Pennsylvania related to these programs. Passage Bio will assist in the transition through a cost-based transition services agreement.

Will Chou, M.D., CEO of Passage Bio, expressed enthusiasm about the out-licensing arrangement, emphasizing its alignment with the company's strategic focus on advancing their lead asset, PBFT02, aimed at treating multiple adult neurodegenerative diseases. Chou highlighted that the transaction would enable Passage Bio to extend its cash runway, affording the company more time and resources to generate substantial clinical data for PBFT02 and explore its potential across additional patient populations.

In a complementary move, Passage Bio has entered a strategic research, collaboration, and license agreement with GEMMA Biotherapeutics. Under this accord, GEMMABio will carry out preclinical and IND-enabling activities for Passage Bio’s research programs, including efforts previously managed by the University of Pennsylvania’s Gene Therapy Program, focusing on Huntington’s disease. Moreover, GEMMABio will offer Passage Bio options to launch new research initiatives targeting four novel central nervous system (CNS) conditions.

Dr. James M. Wilson, co-founder of GEMMABio, conveyed excitement about advancing the three licensed therapies, each addressing severe pediatric diseases. Wilson also emphasized the potential of ongoing collaboration with Passage Bio to leverage joint expertise in CNS research, aiming for significant impacts on patient lives globally.

As part of its agreements with GEMMABio, Passage Bio has revised its strategic collaboration with the University of Pennsylvania’s Gene Therapy Program. Effective July 31, 2024, Passage Bio will cease its preclinical research programs, terminate options for future CNS research programs, and stop funding the discovery research program.

The collective outcome of these agreements is projected to extend Passage Bio's operating cash runway to the end of the second quarter of 2026.

Passage Bio is committed to the development of innovative genetic therapies targeting the root causes of neurodegenerative diseases, with a primary focus on its leading product candidate, PBFT02. PBFT02 is designed to treat neurodegenerative conditions by increasing progranulin levels, thereby restoring lysosomal function and slowing disease progression.

GEMMA Biotherapeutics is dedicated to advancing research and global access to transformative therapies for rare diseases, fostering innovation to expedite the availability of these critical treatments for affected populations.

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