Nonprofit CureDuchenne is Encouraged to See Gene Editing for Duchenne Advance Through Vertex’s Acquisition of Exonics Therapeutics

Deal will accelerate drug development for Duchenne muscular dystrophy, a rare disease that affects 300,000 boys and young men worldwide

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–CureDuchenne, a nonprofit global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (DMD), is enthusiastic with today’s announcement that Exonics Therapeutics, a research company focused on CRISPR/Cas-9 technologies to treat Duchenne muscular dystrophy, has been acquired by Vertex. The acquisition by Vertex, a successful and well-respected biotech company, validates CureDuchenne’s venture philanthropy business model of funding early stage science to advance promising Duchenne research projects.

CureDuchenne co-founded Exonics in 2017 with Dr. Eric Olson, one of the world’s leading CRISPR/Cas-9 scientists. In 2018, Dr. Olson published research that showed a 50% increase of dystrophin in skeletal muscle and 90% increase in the hearts of the canine model.

“Our single focus at Vertex is to bring transformative medicines to people with serious diseases including Duchenne and DM1,” said Dr. Jeffrey Leiden, M.D., Ph.D., chairman, president and chief executive officer of Vertex. “We are thrilled to work with Exonics and the entire Duchenne community to advance the promising science that Eric and his team have developed to change the course of this devastating disease.”

Duchenne muscular dystrophy is the most common and severe form of muscular dystrophy that primarily affects boys. The genetic disease causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Approximately 15,000 U.S. patients are affected with Duchenne, with a total of 300,000 patients worldwide.

“CureDuchenne believed in the science and our team early on and enabled Exonics to show significant proof of principle for gene editing in Duchenne. We are thrilled to work with a large biotech company that can help us move our science from preclinical to clinical as fast as possible,” said Dr. Eric Olson, founder and chief scientific advisor, Exonics Therapeutics.

By investing in promising cutting-edge science, CureDuchenne has de-risked multiple investments, which have attracted biotech and pharmaceutical companies, as well as venture capital firms. With five successful exits since it was founded in 2003, the nonprofit has been able to leverage over $1.5 billion from follow-on investments. This includes early funding to Sarepta Therapeutics at a critical time which then resulted in the approval of the first FDA-approved drug for Duchenne; investment in Nationwide Children’s research in U7 AAV delivered exon skipping, which was recently licensed to Audentes for clinical trials in exon skipping of duplication 2 and development of exon 51 and 53; funding of Bamboo Therapeutics in 2016 that was later acquired by Pfizer; and today’s announcement of Vertex acquiring Exonics.

“We could not be more thrilled about the partnership between Exonics and Vertex. Vertex has an incredible track record in accelerating drug development for rare diseases and they are exactly what the Duchenne community needs to develop and test gene-editing treatments for Duchenne as quickly as possible,” said Debra Miller, founder and CEO, CureDuchenne.

“This announcement gives the entire Duchenne community a reason to celebrate because we are getting closer to a cure for Duchenne. We are grateful to Dr. Eric Olson and the team at Exonics for their hard work in achieving impactful results in their preclinical research and we look forward to continued and expedited successes with Vertex,” continued Miller.

To learn more about CureDuchenne and learn how you can help patients suffering from this debilitating disease, please visit them online at

About CureDuchenne CureDuchenne is the nation’s leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys living today. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. For more information on how to help raise awareness and funds needed for research, please visit, and follow us on Facebook, Twitter and YouTube.

# # #

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.