CRISPR/Cas9 Startup Exonics Therapeutics Launches in Boston

CRISPR-Cas9 Startup Exonics Therapeutics Launches in Boston February 27, 2017
By Alex Keown, Breaking News Staff

BOSTON – New company Exonics Therapeutics came out of the gate with $5 million in seed financing from CureDuchenne Ventures, to harness the power of CRISPR-Cas9 genome editing technology to permanently correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases.

The new company will advance preclinical work done by Eric Olson, Exonics scientific founder and chief science officer. Olson is a professor of molecular biology at the University of Texas Southwestern Medical Center who has researched muscle development, stem cell technologies and transcriptional regulation, according to his university profile page.

Olson’s research at UT Southwestern 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, a protein that helps stabilize and protect muscle fibers,” Exonics said in a statement this morning. Dystrophin is the key protein missing in boys with Duchenne. Company data suggest using this approach could be a permanent treatment for up to 80 percent of children suffering from Duchenne. Additional preclinical data regarding this approach is expected to be released in March, the company said.

“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,” Olson said in a statement.

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. Most patients are diagnosed by the time they reach five years of age and live until their mid-20s.

Cambridge-based Sarepta Therapeutics became the first company to win FDA approval for a DMD treatment called Exondys51, which targets a specific genetic mutation of the drug found in about 13 percent of patients. Illinois-based Marathon Pharmaceuticals won approval for its DMD drug Emflaza earlier this month, to treat patients five years of age and older regardless of genetic mutation. However, that company has put its commercialization plans on hold following an outcry over the $89,000 price tag the drug has.

Olson told Endpoints that the gene-editing direction of Exonics therapy, although still in animal testing phase, would have a higher impact than currently approved therapies. Although currently tested on mouse models, Olson told Endpoints the humanized mice are good indicator of future success” that could be “leagues ahead” of the recently approved DMD treatments.

Exonics will be helmed in the interim by Jak Knowles, the managing director of CureDuchenne Ventures and vice president of medical and scientific affairs at CureDuchenne.

“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,” Knowles said in a statement.

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