Exonics to develop a therapeutic approach to correct underlying genetic mutations in Duchenne, a degenerative genetic disorder without effective treatment options to halt progression of disease.
Exonics Therapeutics, Inc., a newly formed biotechnology company focused on developing gene editing technologies like CRISPR/Cas9 to permanently correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, today announced a commitment of $5 million in seed financing from CureDuchenne Ventures, LLC, a subsidiary of the nonprofit CureDuchenne. The initial seed funding will allow Exonics to advance the preclinical research of its scientific founder and chief science advisor Eric Olson, PhD.
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 the disease. Additional preclinical data is expected to be published in March 2017.
“This represents the next generation of potential Duchenne muscular dystrophy therapies. By leveraging the revolutionary CRISPR/Cas9 method to permanently correct errors in the DNA sequence, it is our hope that we can develop a one-time therapy that provides lifelong benefit to 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 (UTSW), from which Exonics’ technology is licensed.
Duchenne is a rare X-linked genetic progressive muscle disease affecting nearly 15,000 boys in the U.S. and more than 300,000 boys worldwide. There is no cure for Duchenne. Children with the disease start missing development milestones at age 3 and often lose their ability to walk by age 12. All Duchenne patients will suffer from reduced mobility and independence, and ultimately respiratory or cardiac failure results in a reduced life expectancy in the mid-20s.
“We are building on a scientific program of exceptional quality from Dr. Olson’s laboratory with the support of our funder CureDuchenne Ventures, an organization with deep understanding of this disease, therapeutic landscape and large unmet need. This funding underscores their recognition of the potential of Exonics’ gene editing method to develop a novel therapy for Duchenne,” said Cristina Csimma, PharmD, MHP, Executive Chair of the Board of Directors of Exonics, former President and Chief Executive Officer of Cydan, and long-time drug development leader. “Exonics is extremely well-positioned to advance our preclinical program as we follow a rigorous approach to enable translation to clinical trials.”
“We look forward to working closely with the Duchenne community as we aggressively advance gene editing technology to address the significant unmet need for a curative therapy that would dramatically improve the lives of patients with Duchenne and their families,” said Jak Knowles, MD, President and Interim Chief Executive Officer of Exonics. “We are honored to advance the groundbreaking work of Dr. Olson’s laboratory and are eager to translate this approach into an important therapy for the Duchenne community.” Dr. Knowles will continue to serve the Duchenne community as Managing Director of CureDuchenne Ventures, and VP of Medical and Scientific Affairs at CureDuchenne.
“We are delighted to support Dr. Olson and the Exonics team as they advance novel gene editing technology toward a potential cure for Duchenne. CureDuchenne Ventures’ unique model enables us to share our significant scientific resources, expertise and deep connections with the Duchenne community to accelerate scientific breakthroughs, such as Exonics’ program,” said Debra Miller, President of CureDuchenne Ventures and Founder and Chief Executive Officer of CureDuchenne.
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 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 www.exonicstx.com.
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 projects that have advanced to human clinical trials.