First Duchenne Muscular Dystrophy Patient Dosed in Systemic Microdystrophin Gene Therapy

HACKENSACK, N.J., Jan. 17, 2018 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced that the first Duchenne 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.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births.

This $2.2 million grant marks the largest single award in PPMD's almost 23 years. PPMD has a long history of supporting early-stage, innovative research, providing funding at a critical moment in a therapy's development. Gene therapy has been explored for years as a possible therapeutic approach to treating Duchenne. Only recently, though, have advances in science and technology made it seem like a viable treatment option for Duchenne.

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 Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, 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 and PPMD hopes to learn more at the organization's Annual Conference in Arizona this June.

The PPMD grant will help support the manufacturing and clinical costs of the study for trial participants. The award is milestone driven, based on regulatory interactions, vector manufacturing, and patient dosing and follow-up. Last fall, Dr. Mendell and Dr. Rodino-Klapac had successful regulatory interactions laying out the path to bring this study to clinical trial and vector manufacturing is on schedule.

The $2.2 million grant to Nationwide Children's Hospital, Dr. Mendell, and Dr. Rodino-Klapac was made possible in part by the generous support of additional Duchenne foundations, including: Team Joseph, Team Saij, The Fund for Pete's Sake, Rashad's family, and the Nicholoff family. PPMD is grateful for their partnership.

To learn more about PPMD's research investments, please visit the website.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube. 

SOURCE Parent Project Muscular Dystrophy

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