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GLA-GMAC
- Development stage
- Preclinical
- Lead developer
- CellGenTech
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Subcutaneous
01
Overview
GLA-GMAC is an autologous *ex vivo* gene therapy being developed by CellGenTech for the treatment of Fabry disease, a rare lysosomal storage disorder. The therapy utilizes the proprietary GMAC (Genetically Modified Adipocytes) platform, which involves harvesting a patient's own adipocytes (fat cells) and genetically modifying them using viral vectors to express a functional version of the alpha-galactosidase A (α-GLA) enzyme. These modified cells are then reimplanted into the patient, where they are intended to provide a continuous, long-term endogenous source of the enzyme. This approach is designed as a potential 'one-and-done' alternative to chronic enzyme replacement therapy (ERT). The program is currently in preclinical development and is being developed in collaboration with Kyorin Pharmaceutical and Kyowa Kirin.
- Other names
- alpha-galactosidase A gene therapyGMAC-GLA
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