Jesse’s Journey is pleased to share the news that Dystrogen Therapeutics Corporation, a clinical-stage company developing a dystrophin replacement approach using chimeric cell therapy, announced the dosing of the first patient in their Phase 1 human pilot clinical study of DT-DEC01, Dystrophin Expressing Chimeric cells, or DEC, for the treatment of Duchenne muscular dystrophy (DMD). Screening and enrollment of patients are expected to continue over the course of the next 12 months in the European Union, with data from this trial expected once patients have been evaluated for one full year post-treatment. We are excited to see new developments and approaches to treating Duchenne and will continue to monitor the process of DT-DEC01.
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DT-DEC01 is a chimeric cell therapy. The advanced therapeutic medicinal product (ATMP) is made using Dystrogen’s proprietary cell engineering technology which creates a DEC cell. Clinically, DEC cells have been shown to express CD56 at significantly higher levels than myoblasts from Duchenne patients. DEC cells express favorable HLA characteristics which carries multiple advantages. In preclinical studies, DEC cells have also been shown to express clinically significant levels of dystrophin when compared to controls. DEC cell therapy demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and associated function in preclinical trials. Because DEC therapy is designed to prevent triggering an immune system response, a major advantage of DEC therapy is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore involves no risk of off target mutation, does not use viral vectors, and its use is not dependent on the genetic mutation of the DMD patient, thus making DEC a universal therapy for all DMD patients.