First patient dosed in microdystrophin gene therapy in US

Parent Project Muscular Dystrophy (PPMD) announced that the first patient has been dosed with microdystrophin gene therapy by Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, and their team at Nationwide Children’s Hospital. This trial was funded in part by a $2.2 million grant from PPMD in early 2017 as part of the organization’s Gene Therapy Initiative.

“Action Duchenne are delighted to see the progress in gene therapy, specifically microdystrophin therapy, with the advent of new and emerging international clinical trials by different academic groups and companies using different vectors and approaches. Action Duchenne originally identified gene therapy as it’s top priority in 2013 and are continuing to fund this progress with the UNITE-DMD project with other fellow charities.”

According to PPMD founding President and CEO, Pat Furlong, “This is a monumental day in the Duchenne community, a day that 24 years ago when we started Parent Project Muscular Dystrophy, we didn’t think possible. We are so grateful to the drive and passion of Dr. Mendell, Dr. Rodino-Klapac, and the Nationwide Children’s team. We will learn a lot from this study, including answers to questions around the production of sufficient virus, understanding and preventing an immune response, and how to deliver gene therapy systemically. But for now, we are celebrating the first dosing of a Duchenne patient with microdystrophin gene therapy and we are celebrating the bravery of the little boy and his family participating in this breakthrough trial.”

“Bringing this to clinical trial has been an extended process working with a team of researchers at Nationwide Children’s Hospital,” according to Dr. Mendell and Dr. Rodino-Klapac. “The laboratory studies were guided by a careful hand in validating the potential for efficacy for adeno-associated virus delivery in clinical trial. The vector manufacturing facility was responsible for bringing a safe virus carrying the micro-dystrophin gene to the clinic. The regulatory team conveyed all of the proof of principle studies and careful safety data to the RAC, IRB, and FDA, allowing this clinical trial to move forward. The first injection of the virus carrying a modified DMD gene to clinical trial, made over a decade of research a gratifying experience.”

Last year, Sarepta provided monetary and other support to this project through a research and option agreement with Nationwide Children’s.

“After years of dedicated and expert work by Nationwide Children’s Hospital and Drs. Mendell and Rodino-Klapac, and with the support of PPMD, we stand at a potentially transformational moment in our quest to profoundly alter the course of DMD and create a brighter future for these children,” said Douglas Ingram, Sarepta’s president and chief executive officer.

Mr. Ingram continued, “Sarepta is the world leader in precision genetic medicine to treat DMD, spanning RNA-targeted therapies, gene therapy, and gene editing. In service of our mission to profoundly improve the lives of those with DMD, we look forward to the preliminary results from this study by mid-2018 and, results permitting, stand ready to aggressively invest in bringing this therapy to reality and to the DMD community.”

The young patient continues to do well. It is believed that it takes several days for the virus to deliver the payload and fully integrate into heart and muscle cells. He will of course be closely monitored.

For full updates on all research into Duchenne, pre-register for the Action Duchenne International Conference.

Contact Director of Research, Neil Bennett with any questions neil@actionduchenne.org