Published On: Tue, Feb 28th, 2017

Potential Duchenne Muscular Dystrophy therapy from Exonics gets $5m boost

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US based Exonics Therapeutics has secured $5 million seed funding from CureDuchenne Ventures, a subsidiary of nonprofit DMD advocacy group, CureDuchenne to advance its therapy for Duchenne Muscular Dystrophy, a severe form of muscular dystrophy.

Headquartered in , Massachusetts, Exonics Therapeutics is a newly founded biotechnology company that develops gene editing technologies such as CRISPR/Cas9. These technologies permanently rectify most of the mutations that are behind Duchenne muscular dystrophy as well as other neuromuscular disorders.

CureDuchenne’s seed funding will enable the Boston biotechnology company to take the preclinical research of Eric Olson, its scientific founder and chief science advisor to the next level.

Muscular Dystrophy generic image

Muscular Dystrophy. Photo courtesy of Stuart Miles/Freedigitalphotos.net.

Jak Knowles, MD, President and Interim Chief Executive Officer of Exonics said: “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.

“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.”

Above : Histopathology of Duchenne muscular dystrophy, Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells. Image courtesy CDC/Dr. Edwin P. Ewing, Jr, 1972. (Photo by Smith Collection/Gado/Getty Images).

Dr. Olson’s laboratory used adeno-associated virus (AAV) and CRISPR/Cas9 technology to detect and rectify exon mutations in the dystrophin gene. These exon mutations are responsible in inhibiting dystrophin production.

Dystrophin is an essential protein for stabilizing and protecting our muscle fibres.

It is this important protein that is not found in boys suffering from Duchenne muscular dystrophy. Preclinical data that has been published relating to the Dr. Olson’s lab’s research suggest that the novel approach could potentially cure up to 80% of suffering from duchenne muscular dystrophy.

Debra Miller, President of CureDuchenne Ventures who is also the Founder and Chief Executive Officer of CureDuchenne said: “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.”

The genetic progressive muscle disease, Duchenne muscular dystrophy affects around 15,000 boys in the US and over 300,000 boys globally. Presently, there is no treatment for Duchenne muscular dystrophy.

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