GEMMA Biotherapeutics

GEMMA Biotherapeutics is a newly launched company focused on accelerating the development and global access to advanced AAV-based gene therapies for rare diseases. The company recently acquired three clinical-stage pediatric lysosomal storage disorder programs from Passage Bio—PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy—and entered into a strategic collaboration to advance additional CNS genetic medicine programs such as Huntington’s disease. The company is led by renowned gene therapy pioneer Dr. James M. Wilson.[6][8][9] Clinical results are not yet published under the new entity; prior data from Passage Bio indicate ongoing or completed early-phase trials.