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Muscular Dystrophy Association Announces Recipient of 2024 MDA Legacy Award for Achievement in Research, is Jeffrey Chamberlain, Ph.D., Leading Professor in Gene Therapy

NEW YORK, January 30, 2024 – The Muscular Dystrophy Association (MDA) today announced Jeffrey Chamberlain, Ph.D., a leading professor in gene therapy focused on Duchenne muscular dystrophy (DMD) at the Institute for Stem Cell and Regenerative Medicine at the University of Washington, will receive the 2024 MDA Legacy Award for Achievement in Research, for his achievements in translational research.

The award will be presented at the 2024 MDA Clinical & Scientific Conference on Monday, March 4, by Louis Kunkel, PhD, of Boston Children’s Hospital. The MDA Legacy Award for Achievement in Research is an annual recognition for outstanding accomplishments in neuromuscular disease research. This year’s award is for achievement in translational research. The presentation will take place in-person in Orlando, Florida and via live stream. Registration is open for both in-person and virtual registration here. View the full agenda here.

"The Muscular Dystrophy Association is elated to celebrate Dr. Chamberlain and honor him with the 2024 MDA Legacy Award for Achievement in Research for his achievements in translational research. Dr. Chamberlain has been committed to gene therapy from the beginning," said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. "He received our first ever gene therapy award 25 years ago and has continued making major contributions ever since, including the development of the miniaturized gene used in several ongoing clinical trials for Duchenne muscular dystrophy. Just last year we funded a new study for Dr. Chamberlain to make this miniaturized gene product less visible to the immune system and, in a separate project, to develop a way to deliver a full-sized gene for Duchenne. His work is critically important to everything we do in our research program at Muscular Dystrophy Association." 

"It's a true honor to receive the 2024 MDA Legacy Award for Achievement in Research for my work in translational research and a real testament to the continuous support I’ve received from Muscular Dystrophy Association over the past three decades," said Dr. Chamberlain. "I received my very first grant from the Muscular Dystrophy Association – they've been with me from the start and continue to fund my research and serve as a trusted resource. This award is one of the highlights of my career without question. I grew up watching the MDA Telethon when I was a little kid, and I was always intrigued by the idea of muscular dystrophy. After almost 40 years, I am developing gene therapies for Duchenne muscular dystrophy, and we’ve seen real progress and results. Receiving this reward is really a culmination of many years of dedicated work. I’m excited about the 2024 MDA Clinical & Scientific Conference and all we’ll learn and share as a community and all the developments and achievements that are to come."

MDA most recently awarded Dr. Chamberlain with a $200,000 grant for a two-year study on expression of enhanced dystrophins via Adeno-associated viruses (AAV) to circumvent immune responses linked to a subpopulation of DMD patients. The goal is to develop modified dystrophin therapies that could be used in clinical trials and ultimately allow the broad enrollment of patients regardless of their underlying genetic mutation. He was also awarded a grant for $337,000 (made possible by the longstanding annual MDA Edgar Martinez Golf Classic), to deliver dystrophin proteins at least double the size of micro-dystrophins, and combine their use with recently described myotropic AAVs that can be used at lower doses and are more efficient than current AAVs.

As the largest US gathering focused solely on neuromuscular disease, the 2024 MDA Clinical & Scientific Conference will explore the latest research advancements and clinical achievement in the field, with concurrent clinical and scientific sessions. This year's conference will look at advances in neuromuscular disease. The previous recipient of the MDA Legacy Award for Achievement in Research in 2023 was Merit Cudkowicz, MD, MSC Massachusetts General Hospital, for her achievements in clinical research.

Watch the video announcement here.

About Jeffrey Chamberlain, Ph.D.

Jeffrey S. Chamberlain, Ph.D. is the President of the American Society for Gene and Cell Therapy, and Professor and McCaw Chair in Muscular Dystrophy, Director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center Depts. Of Neurology, Medicine and Biochemistry at University of Washington School of Medicine.

The Chamberlain Laboratory studies mechanisms leading to the muscular dystrophies, the structure & function of dystrophin (mutated in DMD, among the most common inherited diseases), and approaches to therapy. The Chamberlain group have performed extensive structure/function studies on dystrophin and on how the dystrophin-glycoprotein complex is assembled in striated muscles, and have pioneered methods for gene delivery to skeletal and cardiac muscles. The Chamberlain group developed miniaturized dystrophin genes that we named “micro-dystrophin” and were also the first to show that AAV vectors could be used for systemic gene delivery to muscle. Significant amelioration of muscular dystrophy has been achieved in animal models for DMD using systemic AAV delivery of micro-dystrophins, or by delivery of CRISPR/Cas9 components to induce gene editing. Several groups are currently planning or conducting human gene therapy trials using Chamberlain lab micro-dystrophins, including Solid Biosciences, Sarepta and Genethon. Learn more here.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.