Scientists from Dystrogen Therapeutics Corp. published data supporting cardioprotective effects of the Companys therapy for muscular dystrophy disorders. Cardiomyopathy is the most devastating cause of morbidity and mortality in Duchenne Muscular Dystrophy (DMD) patients and affects 30% of patients by 14years of age and 50% of patients by 18years of age. Heart failure in these patients is the result of cardiac myocyte death and fibrosis, leading to both diastolic and systolic dysfunction.

Dystrogen Therapeutics Corp has developed an engineered chimeric cell therapy which has been previously shown to restore muscle function in pre-clinical studies. For Duchennes muscular dystrophy, the company has developed dystrophin expressing chimeras DECs. Using the companys proprietary technology, DECs are created by an ex vivo fusion of allogeneic human myoblast from a healthy donor with autologous human myoblast received from DMD patient. DECs have been shown to maintain the ability to express normal dystrophin protein in previously published pre-clinical studies.

The new study published in theOctober 15th, 2019online edition of the journalStem Cell Reports and Reviewsconfirmed the protective effect of DEC on cardiac function after intraosseous delivery shown by increased values of both ejection fraction and fractional shortening, which at 90days revealed a rebound effect when compared to the vehicle injected controls and mice receiving not-chimeric cell therapy. Moreover, these functional improvements correlated with restoration of dystrophin expression in cardiac muscle at 90days post-DEC treatment.

These findings are potentially significant for the treatment of DMD, said Dr. Maria Siemionow, MD, PhD Dystrogen Therapeutics Corp chief scientific officer and the therapys inventor. This study establishes DEC as a promising new option for cardiac protection and potential amelioration of DMD related cardiac pathology.

These data add to the growing body of literature supporting the potential of our chimeric cell platform to restore systemic muscle function, with less potential side effects then gene therapy-based approaches, said Dr. Kris Siemionow, MD, PhD Dystrogen Therapeutics Corp CEO. We are very pleased to have these data published in a highly relevant journal for the field and look forward to further exploring this opportunity.

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Dystrogen Therapeutics Announces That Treatment With DEC Cells Improves Cardiac Function Cardiology2.0 - Cardiology2.0