Cure Duchenne Ventures LLC Provides Funding to RASRx to Help Advance Novel Treatment for Duchenne Muscular Dystrophy

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–CureDuchenne Ventures LLC, a venture philanthropy company that funds
research to find a cure for Duchenne
muscular dystrophy
, announced today that they have invested in
RASRx, a start-up company dedicated to developing therapies for rare
diseases. RASRx is developing an oral therapy that has shown positive
effects on muscle function in a mdx mouse model of Duchenne,
demonstrating improved muscle strength and regeneration, with decreased
muscle inflammation and fibrosis. CureDuchenne
Ventures’ financial support will enable the initiation and acceleration
of critical preclinical development studies and manufacturing. In
addition to financial support, Jak Knowles, Managing Director of
CureDuchenne Ventures, will join the RASRx Board of Directors.

“Duchenne is a complex disease and we have has been working to treat
the whole disease
with a multi-pronged approach to find treatments
for the many effects that Duchenne has on the body,” said Debra Miller,
president of CureDuchenne Ventures. “We are delighted to partner with
RASRx and their dedicated team on this novel approach that could become
a significant avenue for treatment for Duchenne.”

Duchenne
is a fatal genetic disease that causes muscles to degenerate. It impacts
approximately 1 in 3,500 boys. Those with Duchenne are usually diagnosed
by age 5, lose their ability to walk by 12 and most don’t survive their
mid-20s. There is currently no approved treatment or cure for Duchenne.

“We are passionate about making an impact in healthcare in areas of
unmet needs like Duchenne muscular dystrophy,” said Kathleen Rodgers, a
founder of RASRx. “Our lead program at RASRx, which has previously
received DoD
funding
, takes a mutation independent approach to slowing and
potentially reversing disease progression to bring relief to patients
with Duchenne, and possibly other muscular dystrophies. We are thrilled
by the potential of this relationship as CureDuchenne’s knowledge of the
disease and familiarity with the needs of the patients are invaluable as
we move forward in the development of this promising treatment.”

CureDuchenne Ventures LLC was formed by CureDuchenne, a national
non-profit that has funded seven research projects that have advanced to
human clinical trials. CureDuchenne has leveraged $100 million in pharma
and biotech research and development investments. Now three of the
projects CureDuchenne supported with funding, BioMarin Pharmaceutical,
Sarepta Therapeutics and PTC Therapeutics, are the closest to becoming
the first drugs to be approved for the treatment of the disorder.

About CureDuchenne Ventures

CureDuchenne Ventures LLC collaborates with pharmaceutical and
biotechnology companies to facilitate the development of drugs to treat
Duchenne muscular dystrophy. CureDuchenne Ventures LLC was formed by
CureDuchenne, a national nonprofit that has a successful track record of
supporting research and raising awareness of the disease.

About CureDuchenne

CureDuchenne was founded in 2003 with a focus on saving the lives of
those with Duchenne muscular dystrophy, a disease that affects more than
300,000 boys worldwide. With support from CureDuchenne the FDA could
approve three pharmaceutical treatments within the next year. These
treatments may lessen the effects of the disease for those with certain
mutations of Duchenne, but there is still much to be done to find a
cure. For more information, please visit CureDuchenne.org
and follow us on Facebook,
Twitter
and YouTube.

About RASRx

RASRx will develop and commercialize technology licensed from the
University of Southern California. The founders of the company have
extensive drug development expertise having taken projects through late
clinical stage development. RASRx has innovated a leading-edge, systems
biology approach to treating the complex muscle pathology of Duchenne
muscular dystrophy.

Contacts

CureDuchenne
Karen Harley, 949-872-2552