NEW YORK – Passage Bio has licensed out three of its gene therapies for pediatric lysosomal storage diseases to the newly formed Gemma Biotherapeutics, the company announced Thursday.
Under the terms of the agreement, GemmaBio will purchase supplies of clinical products from Passage Bio for $10 million and pay another $10 million once it attains certain business milestones. Passage Bio is also eligible to receive $114 million in additional development and commercial milestone payments and royalties in exchange for sublicenses to intellectual property and the transfer of clinical trial materials and product supply.
In the deal, GemmaBio garners exclusive worldwide rights to develop and potentially commercialize Passage Bio's GM1 gangliosidosis gene therapy candidate PBGM01, its Krabbe disease gene therapy asset PBKR03, and the metachromatic leukodystrophy gene therapy PBML04. Passage Bio will provide support as its programs transition to GemmaBio, which will take over Passage Bio's remaining financial obligations to the University of Pennsylvania for the licensed programs.
GemmaBio, which launched Wednesday, is cofounded and led by Passage Bio cofounder James Wilson. Wilson announced he would step down from his role as director of UPenn's Gene Therapy Program on Wednesday in connection with the launch of GemmaBio and a second company, the contract research organization Franklin Biolabs, which is to take up some of the UPenn program's functions and translate discoveries into commercial treatments.
Philadelphia-based Passage Bio expects the funds from the licensing agreement with GemmaBio to extend its operating cash runway to the end of Q2 2026. "This transaction is a key initiative in fulfilling our strategy to focus on advancing our lead asset, PBFT02, in multiple adult neurodegenerative diseases while continuing our preclinical research in high potential [central nervous system] indications," Passage Bio President and CEO Will Chou said in a statement.
Separately, Passage Bio and GemmaBio inked a research, collaboration, and license agreement, under which GemmaBio will conduct preclinical and investigational new drug (IND)-enabling activities for certain Passage Bio research programs, including Huntington's disease, which was previously managed by UPenn's Gene Therapy Program. GemmaBio will also provide Passage Bio with options to initiate new research programs in four CNS indications.
As a result of these agreements, Passage Bio amended its strategic collaboration with UPenn's Gene Therapy Program and discontinued preclinical research efforts, as well as terminated its remaining options to advance future CNS research programs and fund the discovery research program.