GAD65-CAR-Tregs

GAD65-CAR-Tregs is an investigational cell therapy consisting of T regulatory cells (Tregs) engineered with a chimeric antigen receptor (CAR) specific for the 65 kD isoform of glutamic acid decarboxylase (GAD65). GAD65 is a key autoantigen involved in the pathogenesis of type 1 diabetes (T1D) and latent autoimmune diabetes in adults (LADA). By targeting GAD65, these CAR-Tregs are designed to selectively home to the pancreatic islets, where they encounter the cognate antigen and undergo activation and proliferation. Once activated, the GAD65-CAR-Tregs exert localized immunosuppressive effects, including the suppression of cytotoxic T lymphocytes (CTLs) and other diabetogenic T cells, thereby protecting pancreatic beta cells from autoimmune destruction. Preclinical data from humanized mouse models and ex-vivo human pancreatic tissue studies suggest that GAD65-CAR-Tregs can reverse T1D pathophysiology, normalize metabolic parameters such as fasting blood glucose and insulin levels, and facilitate the recovery and reconstitution of pancreatic islets.

Other names

GAD65-CAR-T regulatory cellsGAD-65-CAR-T regulatory cellsGAD 65-CAR-T regulatory cellsGlutamic Acid Decarboxylase 65-specific CAR-Tregs