Drug intelligence / Profile preview

FIX-GMAC

Development stage
Preclinical
Lead developer
CellGenTech
Modality
Cell Therapies, Gene Therapies
Administration
Implant
01

Overview

FIX-GMAC (also known as HB-GMAC) is an investigational autologous cell and gene therapy being developed by CellGenTech for the treatment of hemophilia B. The therapy utilizes CellGenTech's Genetically Modified Adipocytes (GMAC) platform, where pre-adipocytes are isolated from a patient's own subcutaneous fat tissue and genetically modified ex vivo using a viral vector to express the normal human coagulation factor IX (FIX) gene. Once implanted back into the patient, these modified adipocytes are intended to continuously secrete functional factor IX, providing a sustained, long-term source of the clotting factor to prevent bleeding episodes. FIX-GMAC is currently in the feasibility and pre-preclinical stages of development.

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