Sharon Hesterlee, PhD, on Collaborating to Expand Therapy Development to More Dystrophies


The chief research officer of the Muscular Dystrophy Association discussed the need for disease-modifying therapies in other muscular dystrophies and programs the association has announced to this end.

“There are so many rare diseases that don't have big commercial markets. We see a lot of activity [with muscular dystrophies], it's been growing exponentially around Duchenne and some of the more common ones. But we have this big portfolio of dystrophies. So we've launched an internal program that we call MDA Kickstart, and we basically are operating it like a gene therapy company. We're working with the University of Florida on an ultra-rare indication, and we’ll hopefully make a big announcement about the indication in a month or 2.”

2023 has yielded unprecedented progress in the treatment landscape of Duchenne muscular dystrophy (DMD) with the summer approval of delandistrogene moxeparvovec (marketed as Elevidys by Sarepta), the field’s first gene therapy.1 Despite the progress in DMD, there remain many muscular dystrophies without disease modifying therapies (DMTS) available or in investigation.

CGTLive spoke with Sharon Hesterlee, PhD, chief research officer, Muscular Dystrophy Association (MDA) to learn more about how the MDA is working to make gene therapies available to a wider population of patients with muscular dystrophies. She noted that collaboration between different advocacy groups and between academic centers is important for generating solutions for these patients and highlighted 2 programs born out of collaboration – a recent program at the University of Florida investigating neutralizing anti-AAV antibodies and the MDA Kickstart program seeking to develop DMTs for other dystrophies.2

1. Sarepta Therapeutics announces FDA approval of ELEVIDYS, the first gene therapy to treat Duchenne muscular dystrophy. News release. Sarepta Therapeutics. June 22, 2023.
2. CureDuchenne, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy announce collaborative project to focus on re-dosing gene therapy in Duchenne Muscular Dystrophy. News release. Muscular Dystrophy Association. June 15, 2023. Accessed June 16, 2023.
Related Videos
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics
Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics
Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa
Debora Mazzetti, MS, on Multitargeting MicroRNA in Glioblastoma
Abhishek Gupta, BS, the senior vice president of genetic medicines at Syneos Health
Francesca Del Bufalo, MD, PhD, a medical doctor and scientist at Bambino Gesù Chidren’s Hospital
Luke Roberts, MBBS, PhD, on Early Clinical Data on Congestive Heart Failure Gene Therapy
Lawrence R. Lustig, MD, the chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians
© 2024 MJH Life Sciences

All rights reserved.