Duchenne Muscular Dystrophy

Duchenne Muscular
Dystrophy

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. The therapy is based on ex vivo fusion of allogeneic human myoblast with autologous human myoblast received from the DMD patient. We have demonstrated that 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. This has been shown to be the case in a number of published preclinical studies. DEC therapy minimizes the immune response and the need for immunosuppression since the patient will recognize DEC cells as “self” cells. This new approach is based on the delivery and restoration of dystrophin in affected muscles, with the goal of preventing the premature loss of mobility and early mortality seen in DMD patients.

The therapy will target the entire DMD patient population because this approach 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