CureDuchenne Ventures Invests in New Gene Editing Company Exonics Therapeutics to Advance CRISPR/Cas9 Therapy for Duchenne Muscular Dystrophy

Ventures, a subsidiary of the nonprofit CureDuchenne that funds research
to find a cure for Duchenne muscular dystrophy, announced today that
they have committed $5 million in seed financing in Exonics
, a new biotechnology company focused on utilizing gene
editing technologies like CRISPR/Cas9 to advance the development of a
treatment for Duchenne muscular dystrophy. Exonics’ CRISPR/Cas9
technology is a potential one-time treatment that would make a permanent
correction of the mutation that causes Duchenne.

Exonics Therapeutics will use proceeds from the seed financing to
advance the preclinical research of its scientific founder and chief
science advisor Eric
, PhD, to a clinic-ready therapy. Dr. Olson’s laboratory has
demonstrated the ability to use adeno-associated virus (AAV) to deliver
a payload based on CRISPR/Cas9 technology that can identify and correct
exon mutations that prevent the production of dystrophin, a protein that
helps stabilize and protect muscle fibers. Dystrophin is the key protein
missing in boys with Duchenne, and published preclinical data suggest
that this approach has the potential to permanently treat up to 80
percent of children suffering from Duchenne.

Watch this video
for more information about the research being conducted in the Olson
lab, on which Exonics’ technology is based.

“The Duchenne community needs novel and diverse approaches to treat and
cure this devastating disease,” said Debra Miller, Founder and CEO of
CureDuchenne. “Exonics’ CRISPR/Cas9 technology has the potential to
dramatically improve the lives of those who live with Duchenne. We are
hopeful that a one-time treatment with gene editing therapy could
provide a lifelong benefit to Duchenne patients.”

“We are delighted to provide seed funding for Exonics and over the past
year, have worked closely with Dr. Olson and his team, to create a
company which is well positioned to advance promising gene editing
treatments for Duchenne,” said Dr. Jak Knowles, President and Interim
Chief Executive Officer of Exonics, and Managing Director of
CureDuchenne Ventures. “This technology could be a breakthrough for the
Duchenne community because of the ability to use a patient’s own DNA to
fix the mutated gene.”

“The creation of Exonics, in conjunction with CureDuchenne Ventures,
will allow us to work toward a one-time therapy that would make a big
impact on the lives of Duchenne patients,” said Dr. Olson, who also
serves as Professor and Chairman of the Department of Molecular Biology
at the University of Texas Southwestern Medical Center, from which
Exonics’ technology is licensed. “CureDuchenne has strong relationships
with the Duchenne community including patients, families, physicians and
scientists in academia and industry. These relationships, CureDuchenne’s
deep knowledge of Duchenne and all the therapeutic approaches for the
disease; and their strong leadership team make them a great partner to
help accelerate drug development.”

To donate to the CRISPR/Cas9 gene editing research, click here.

About Exonics Therapeutics

Exonics Therapeutics is advancing gene editing technologies like
CRISPR/Cas9 to permanently correct the majority of Duchenne muscular
dystrophy mutations. Preclinical data suggest Exonics’ novel gene
editing approach has the potential to permanently treat up to 80 percent
of children who suffer from Duchenne. Exonics’ technology is licensed
from the University of Texas Southwestern Medical Center (UTSW) and is
based on the research of its scientific founder and chief science
advisor, Eric Olson, PhD. The Company’s corporate office is located in
Boston, Mass., and research activities are being conducted in Dallas,
Tex. For more information, please visit

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 funded nine research
that have advanced to human clinical trials. CureDuchenne
has leveraged more than $1.3 billion in follow-on investment from
pharmaceutical companies and biotech to fund research. CureDuchenne 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.

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, nine research
projects have advanced to human clinical trials. CureDuchenne also is
the innovator bringing physical therapy and standard of care to local
communities around the country through CureDuchenne
. For more information, please visit
and follow us on Facebook,
Twitter, Instagram
and YouTube.


Karen Harley, 949-872-2552