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—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 www.cureduchenne.org.

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 www.cureduchenne.org,
and follow us on Facebook,
Twitter
and YouTube.

Contacts

Matter Communications
Dan Ventresca
617.391.9898
[email protected]