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.
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SOURCE Parent Project Muscular Dystrophy