The venture philanthropy arm of the nonprofit CureDuchenne has committed $5 million in seed financing toward a startup focused on using gene-editing technologies such as CRISPR/Cas9 to permanently correct most mutations causing Duchenne muscular dystrophy (DMD) and other neuromuscular diseases.
CureDuchenne Ventures and Exonics Therapeutics said today that the seed funding will allow the startup to advance into the clinic the preclinical research of its scientific founder and chief science advisor Eric Olson, Ph.D.
Dr. Olson is professor and chairman of the Department of Molecular Biology at the University of Texas Southwestern Medical Center (UTSW), from which Exonics’ technology is licensed. His laboratory has shown 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, the key protein missing in boys with DMD.
Dr. Olson’s lab has published preclinical data suggesting that this one-time treatment approach could potentially be a permanent treatment for up to 80% of children suffering from the disease, with additional preclinical data expected to be published in March.
“This represents the next generation of potential Duchenne muscular dystrophy therapies,” Dr. Olson declared in a statement. “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.”
According to CureDuchenne, DMD affects nearly 15,000 boys in the U.S. and more than 300,000 boys worldwide.
CureDuchenne Ventures has been formed by CureDuchenne, which to date has funded nine research projects that have advanced into human clinical trials. CureDuchenne says its funding has leveraged more than $1.3 billion in follow-on research investment from biopharma companies.
“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,” added Jak Knowles, M.D., managing director of CureDuchenne Ventures, and vp of medical and scientific affairs at CureDuchenne.
Dr. Knowles also serves as president and interim CEO of Exonics, which bases its corporate office in Boston and conducts research activities in Dallas.