DMD Gene Editing Therapy Could Cure Affected Boys

Duchenne muscular dystrophy (DMD) gene editing therapy seems to be the only hope for young boys afflicted with the disease. Scientists are still searching for a therapy that can substantially improve patients’ lifespan and quality if life. Fortunately, a new startup company, the Exonics Therapeutics, looks determined to bring hope to patients by using the cutting-edge CRISPR/Cas9 gene editing technology.

The Boston, Massachusetts startup officially launched on Monday. They have received $5 million in funding from the investment arm of a non-profit DMD advocacy group, CureDuchenne Venture. The newly-launched company will focus on using CRISPR/Cas9 in treating DMD.

DMD's gene editing therapy is currently deemed as the only hope to cure the disease. It is the most common severe form of childhood muscular dystrophy that affects both skeletal and heart muscles of young boys. Left untreated, the patients can lose their ability to walk at around 10 to 12 years old, and are medically expected to die in their mid-20s due to heart failure, the Fierce Biotech reports.

Affecting around 15,000 boys in the U.S., the disease strikes around 300,000 globally. Therapy for the condition was predominantly in the form of steroids, but in September, the FDA approved a new type of treatment called Exondys 51 (eteplirsen) from Sarepta. However, this drug is limited in treating only patients with gene dystrophy mutation, which affects around 13% of the population with DMD, the MedCity News reports.

Talking about DMD gene editing therapy, Dr. Eric Olson, scientific founder and chairman of the Department of Molecular Biology at the University of Texas Southwestern Medical Center, says that CRISPR/Cas9 technology could offer possible cure. The technique identifies and corrects exon mutations that prevent the production of dystrophin, helping stabilize and protect muscle fibers. He says that early preclinical data suggest that the approach can potentially treat up to 80 percent of children with DMD.

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