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GT-GBA1-S05

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

Overview

GT-GBA1-S05 is a preclinical ex vivo hematopoietic stem cell gene therapy being developed by Glafabra Therapeutics for the treatment of Gaucher disease. Utilizing the proprietary Live-cel platform, the therapy involves harvesting a patient's own hematopoietic stem cells and genetically modifying them using lentiviral vectors to carry a functional copy of the GBA1 gene. Once reinfused, these engineered cells engraft in the bone marrow and differentiate into various blood cell lineages that provide a continuous source of the glucocerebrosidase (GBA) enzyme throughout the body via a mechanism known as cross-correction. This one-time treatment is designed to restore enzyme activity and provide a durable therapeutic alternative to traditional enzyme replacement therapy.

Other names: GT-GLA (Gaucher program)

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