Drug intelligence / Profile preview

HB-GMAC

Development stage
Preclinical
Lead developer
CellGenTech
Modality
Cell Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Subcutaneous
01

Overview

HB-GMAC is an autologous *ex vivo* gene therapy being developed by CellGenTech for the treatment of Hemophilia B. The therapy utilizes the proprietary GMAC (Gene Medicine with Autologous Cells) platform, which involves harvesting a patient's own adipocytes (fat cells), genetically modifying them *ex vivo* to express functional Factor IX (FIX) protein, and then reintroducing them into the patient. By using adipocytes as a delivery vehicle, the therapy is designed to provide sustained production of the clotting factor that is deficient or absent in Hemophilia B patients. This approach represents a novel alternative to traditional gene therapies that use viral vectors or hematopoietic stem cells. HB-GMAC is currently in the preclinical stage of development.

02

Targets

F9 (Coagulation Factor IX)

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