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