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About RASRx

RASRx is a start-up company dedicated to developing therapies for rare diseases. The company 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.

RASRx Headquarter Location


United States

Latest RASRx News

RASRx Receives Orphan Drug Designation from FDA

Jun 15, 2017

GlycoMimetics’ GMI-1271 Receives EU Orphan Drug Designation for Acute Myeloid Leukemia Through this ODD, RASRx is eligible for financial incentives that can stimulate investment in this program and expedite its regulatory timeline. In concert with CureDuchenne , the company is now transitioning to toxicology studies to advance RASRx1902 towards human clinical trials for the treatment of Duchenne. RASRx’s founders worked with the USC Stevens Center for Innovation, the technology transfer office for the University of Southern California, to exclusively license RASRx1902. Funding by CureDuchenne Ventures and a collaborative U.S. Department of Defense research grant with USC has accelerated the preclinical development of this program. “Innovation is a core part of USC’s culture and we are proud to support Dr. Kathleen Rodgers and RASRx to facilitate the technology transfer of RASRx1902 for DMD,” said Michael Arciero, J.D. Director of Technology Commercialization and New Venture, USC Stevens Center for Innovation. 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 are limited approved treatments and no cure for Duchenne. “We are passionate about making an impact in areas of unmet needs like Duchenne Muscular Dystrophy,” said Kathleen Rodgers, a co-founder of RASRx. “This lead program at RASRx takes a mutation-independent approach to halting, and possibly reversing, the degenerative progression of Duchenne. Through this approach, we hope to bring relief to patients with Duchenne, as well as other muscular dystrophies. We have been benefitted immensely from CureDuchenne’s knowledge of the disease and familiarity with the needs of the patients. This collaboration has been invaluable as we move forward in the development of this promising treatment.” About RASRx RASRx is developing and commercializing technology licensed from the University of Southern California. The founders of the company are USC researchers with 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. About CureDuchenne CureDuchenne is the leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. CureDuchenne’s mission is to extend and improve the lives of those affected by Duchenne. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne provides the Duchenne community with resources on the best standard of care through its CureDuchenne Cares program. For more information, please visit and follow us on Facebook , Twitter , Instagram and YouTube . About the USC Stevens Center for Innovation The USC Stevens Center for Innovation is the technology transfer office for the University of Southern California and a university-wide resource for USC innovators in the Office of the Provost. The mission at the USC Stevens Center for Innovation is to maximize the translation of USC research into products for public benefit through licensing, collaborations, and the promotion of entrepreneurship and innovation. For more information, visit . View source version on CureDuchenne

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Expert Collections containing RASRx

Expert Collections are analyst-curated lists that highlight the companies you need to know in the most important technology spaces.

RASRx is included in 1 Expert Collection, including Regenerative Medicine.


Regenerative Medicine

1,767 items

Regenerative medicine refers to the process of activating, replacing, engineering or regenerating human genetic material, cells, tissues or organs to restore normal function. It also includes bioengineered tissues used for in vitro testing (e.g. organ-on-a-chip, organoids).

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