Courtney Young, PhD, on Gene Editing Approaches to Treating Neuromuscular Disease

“Gene editing is kind of the next generation of gene therapy because you're editing the patient's own DNA. Some of those advantages are that it results in permanent changes to the DNA, so it's thus longer lasting and can be passed to daughter cells that divide from the targeted cell. You also maintain the endogenous gene promoter and regulatory sequences, so you don't have to rely on exogenous overexpression, like with other types of gene therapy, and then you can retain more of the protein coding sequence.”

With significant unmet needs remaining for patients with Duchenne muscular dystrophy (DMD), many companies are developing genetic medicines for the treatment of these patients. MyoGene Bio’s MyoDys^45-55 is a preclinical gene editing therapy targeted at a DNA region where mutations are found in approximately half of patients with DMD.

In an interview with CGTLive, Courtney Young, PhD, the co-founder and chief executive officer of MyoGene Bio, spoke about the company’s mouse model research on MyoDys^45-55, data from which were presented in the session “Translation of Gene Editing Technologies” at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.

Young noted that in the humanized mouse model, the gene editing approach was capable of restoring expression of the dystrophin protein and improving function. She mentioned that the approach was effective with multiple means of delivery and across multiple time points. Young also discussed some of the advantages of gene editing approaches for treating genetic diseases in general and the revolutionary potential of CRISPR for treating neuromuscular diseases.

Editor’s Note: Young disclosed that she is an owner and board member of MyoGene Bio, chief executive officer of MyoGene Bio, an inventor of patents belonging to MyoGene Bio and UCLA, an inventor of patents belonging to UCLA, and that she has stock ownership in MyoGene Bio and Skygene (startups developing therapies for muscle diseases).