Home / Rare Diseases / Dystrogen Therapeutics Announces that Treatment with Dystrophin Expressing Chimeric Cells Improves Cardiac Function in Preclinical Duchenne’s Study

Dystrogen Therapeutics Announces that Treatment with Dystrophin Expressing Chimeric Cells Improves Cardiac Function in Preclinical Duchenne’s Study

CHICAGO, Nov. 5, 2019 /PRNewswire/ — Scientists from Dystrogen Therapeutics Corp. published data supporting cardioprotective effects of the Company’s 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 14 years of age and 50% of patients by 18 years 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 Duchenne’s muscular dystrophy, the company has developed dystrophin expressing chimeras “DECs.” Using the company’s 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 the October 15th, 2019 online edition of the journal Stem Cell Reports and Reviews confirmed the protective effect of DEC on cardiac function after intraosseous delivery shown by increased values of both ejection fraction and fractional shortening, which at 90 days 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 90 days 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 therapy’s 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.”

About Dystrogen Therapeutics

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing personalized therapies for rare diseases. The company has developed a chimeric cell therapy platform. Dystrophin expressing chimeras “DEC” are based on ex vivo fusion of allogeneic human myoblast derived from donors with autologous human myoblast received from the DMD patient, where chimeric cells maintain the ability to express normal dystrophin protein. DEC cells increase the number/pool of normal myoblasts and reduce inflammation. DEC cells induce replacement of fibrotic tissue, thus significantly improving muscle strength and function in DMD pre-clinical studies. The therapy minimizes immune response effects and the need for immunosuppression. 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. The company is planning on enrolling patients for its DEC chimeric cell therapy Duchenne muscular dystrophy trial. This therapy offers a unique advantage and allows the patient’s body and immune system to accept the chimeric cells without rejection. Pre-clinical results have demonstrated that increased dystrophin levels correlate with improved functional outcomes. First clinical results from DEC therapy are expected in late 2020.

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