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UF Powell Gene Therapy Center is site for Duchenne Muscular Dystrophy clinical trial

Researchers at the University of Florida Powell Gene Therapy Center are part of an innovative clinical trial aimed at repairing hearts damaged by the effects of Duchenne muscular dystrophy.

The HOPE-Duchenne (Halt CardiomyOPathy progrEssion in Duchenne) trial is sponsored by Capricor Therapeutics Inc., a biotechnology company focused on the discovery and development of stem cell therapies for cardiac repair.

Capricor’s investigatory product, CAP-1002, uses donor-derived cardiac stem cells that patients will receive directly into all three coronary arteries, with the goal of reducing scarring, also known as cardiac fibrosis. Published reports about another recent trial using this method, Capricor’s DYNAMIC trial, showed adult patients with Class III heart failure improved.

“We are honored to have been selected by Capricor for this important study,” said Barry Byrne, M.D., Ph.D., director of the UF Powell Gene Therapy Center, a professor of pediatrics at the UF College of Medicine, and a member of the UF Genetics Institute. “DMD is a progressive disorder that is devastating to patients and their families. Therapies that can improve and lengthen the lives of these patients are desperately needed and CAP-1002 promises to treat the leading cause of death in DMD.”

The HOPE-Duchenne trial is designed to enroll 24 Duchenne patients in a randomized, multicenter study evaluating the safety and efficacy of CAP-1002. Heart failure initiated by the formation of extensive scar tissue in the heart is currently the leading cause of death in patients with Duchenne muscular dystrophy. Enrollment in the trial is also taking place at Cincinnati Children’s Hospital Medical Center in Cincinnati, and at Cedars-Sinai Heart Institute in Los Angeles. The Duchenne subjects will be followed for at least a year in order to measure the long-term effectiveness of the potential therapy.

“We are delighted to have the participation of Dr. Byrne and the Powell Gene Therapy Center at the University of Florida in our HOPE-Duchenne clinical trial,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer. “Dr. Byrne is an internationally recognized pediatric cardiologist, and has been relentless in his pursuit of treatments for various forms of rare diseases, including DMD. As a clinical investigator in HOPE-Duchenne, Dr. Byrne’s involvement strengthens Capricor’s program and supports the therapeutic potential of CAP-1002 for patients with DMD cardiomyopathy.”

For more information, please visit: or (NCT02485938) or contact the UF clinical research coordinator, Gee Kim, at 352-294-8283 or

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