CART-GFRa4 cells

CART-GFRa4 cells are an autologous cell therapy product consisting of a patient's own T lymphocytes that have been genetically engineered using a lentiviral vector to express a chimeric antigen receptor (CAR) targeting the glial-derived neurotrophic factor family receptor alpha 4 (GFRα4). The CAR construct includes an extracellular single-chain variable fragment (scFv) specific for GFRα4, fused to intracellular signaling domains from CD3ζ and the co-stimulatory molecule 4-1BB. Upon infusion, these modified T cells recognize and bind to GFRα4-expressing tumor cells—primarily medullary thyroid carcinoma (MTC)—leading to targeted cytotoxicity and cytokine release. Preclinical studies have demonstrated potent antitumor activity in vitro and in vivo against MTC models with high specificity for human tumor tissue. A phase 1 clinical trial is ongoing at the University of Pennsylvania evaluating safety and feasibility in patients with recurrent or metastatic MTC[3][5][7].

Other names

autologous anti-GFRa4 CAR-TCR-zeta-4-1BB-expressing T cellsGFRα4 CAR-T cellsGFRα4-specific CAR Ts