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FVIII-GMAC
- Development stage
- Preclinical
- Lead developer
- CellGenTech
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Implant, Subcutaneous
01
Overview
FVIII-GMAC is an ex vivo cell and gene therapy being developed by CellGenTech for the treatment of hemophilia A. The therapy utilizes CellGenTech's Genetically Modified Adipocytes (GMAC) platform, where pre-adipocytes isolated from the patient's own subcutaneous fat are genetically modified using viral vectors to express the normal human coagulation factor VIII (FVIII) gene. Once implanted back into the patient, these genetically modified adipocytes are intended to continuously secrete functional factor VIII, providing a long-term, stable source of factor VIII replacement to prevent bleeding episodes. FVIII-GMAC is currently in preclinical development.
- Other names
- Factor VIII Gene-Genetically Modified Adipocytes血液凝固第VIII因子遺伝子導入前脂肪細胞
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