Company intelligence / Profile preview
GEMMA Biotherapeutics
Philadelphia, Pennsylvania, United Statesgemmabiotx.com ↗
- Ownership
- Private
- Employees
- ~66
- Development stage
- Preclinical
01
Overview
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.
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- Gene therapy (AAV-based vectors)Biological preparations[1][3][8]
- Industry
- Biotech
02
Drug pipeline
3 assets2 more assets in the full pipeline.
03
Partnerships
Passage Bio, Inc.: Out-license of three clinical-stage pediatric LSD programs; strategic R&D collaboration on CNS indications including Huntington’s Disease[6][9]Franklin Biolabs (close operational partnership; Franklin provides CRO services)[3]Fiocruz/Brazilian Ministry of Health—collaboration on immunobiologics/gene therapies in Brazil[8]
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