GenPHSat

GenPHSat is an autologous, ex-vivo gene-edited cell therapy consisting of primary human satellite cell-derived muscle stem cells. Developed by Dr. Simone Spuler at the Experimental and Clinical Research Center (ECRC), a joint institution of Charité – Universitätsmedizin Berlin and the Max Delbrück Center, the therapy is designed to treat various forms of limb-girdle muscular dystrophy (LGMD). The process involves harvesting muscle stem cells from a patient's own muscle tissue, using CRISPR-Cas9 technology to correct specific genetic mutations in vitro, and then transplanting the edited cells back into the patient's muscle tissue. This approach aims to restore functional protein expression and muscle regenerative capacity in patients for whom no other therapies are currently available. It is being evaluated in the first-in-human Phase 1/2a GenPHSats-bASKet clinical trial.

Other names

GenPHSatsautologous gene-edited primary human satellite cell-derived muscle stem cells