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GT-GAA-S04

Development stage: Preclinical
Lead developer: Glafabra Therapeutics
Modality: Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration: Intravenous

Overview

GT-GAA-S04 is a preclinical ex vivo hematopoietic stem cell (HSC) gene therapy being developed by Glafabra Therapeutics for the treatment of Pompe disease. Utilizing the proprietary Live-cel platform, the therapy involves harvesting a patient's own HSCs and genetically modifying them with a lentiviral vector to deliver a functional copy of the GAA gene, which encodes the acid alpha-glucosidase enzyme. Once reintroduced into the patient, these modified cells engraft in the bone marrow and produce progeny that secrete the functional enzyme. This enzyme is then taken up by affected tissues through cross-correction, potentially providing a durable, one-time treatment alternative to chronic enzyme replacement therapy.

Other names: GT-GLA (Pompe program)

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