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

01/05/2016 | 02:21pm EST

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.


ę Business Wire 2016
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