Duchenne Muscular Dystrophy

Duchenne Muscular

Dystrophin Expressing Chimeric Cells (DEC) based therapy developed by Dystrogen Therapeutics aims to target all patients suffering from Duchenne muscular dystrophy, irrespective of existing genetic mutation. DEC product is based on ex vivo fusion of allogeneic human myoblast derived from close relative donors with autologous human myoblast received from DMD patient, where chimeric cells maintain the ability to express normal dystrophin protein. DEC cells will increase the number/pool of normal myoblasts and will reduce inflammation and induce replacement of fibrotic tissue thus significantly improving muscle strength and function in DMD patients. Our therapy minimizes immune response effect and the need for immunosuppression since the patient will recognize DEC cells as “self” cells. This new approach will be based on delivery and restoration of dystrophin in affected muscles preventing the premature loss of mobility and early mortality of DMD patients.

DEC product will target the entire DMD patient population, because our DEC therapy is not targeting any specific gene mutation and does not require genome editing.

These features qualify DEC therapy as a novel and attractive cell based approach for restoration of muscle function in DMD patients, alone or in combination with other therapies where dosing is limited and sensitization occurs.

Mechanism of Action