Myosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure to improve the quality of life, increase longevity and reduce the disease burden of Duchenne muscular dystrophy (DMD) and other neuromuscular diseases.
Matthew Lumley Matthew Lumley
Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. Currently, Matthew is a Venture Partner at Medicxi.
Nick Whitehead Nick Whitehead Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. His innovative Muscle-Targeted, Non-Viral platform has the potential to provide a novel gene therapy treatment for DMD a wide-range of other neuromuscular and cardiac disorders.
Stan Froehner Stan Froehner Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. He has extensive research experience in DMD. He is currently a Professor of Physiology and Biophysics at the University of Washington. Stan was elected to the Washington State Academy of Sciences in 2013 and selected as a Fellow of the American Association for the Advancement of Sciences in 2017.
Debra Miller Debra Miller Debra Miller is Chief Executive Officer and Founder of CureDuchenne, a non-profit organization dedicated to finding and funding a cure for Duchenne muscular dystrophy, a degenerative neuromuscular disease. She founded CureDuchenne in 2003 after her only son, Hawken, was diagnosed with Duchenne and very little was known about the disease. At the time, there were no available treatments and no hope for a cure.
Seeing a need, Debra got to work raising money. After reaching out to scientists all over the world and logging hundreds of hours of research, CureDuchenne made initial investments into small biotech companies working to accelerate drug development towards a cure. CureDuchenne’s investments into early-stage Duchenne research proved to be an effective catalyst for advancing promising science, attracting additional funds to bring therapeutics through clinical trials and finally to market.
Since Debra’s tireless work began, CureDuchenne has:
- Funded 16 research projects that have advanced to human clinical trials
- Contributed early funding to the first FDA-approved Duchenne drug
- Pioneered the first and only Duchenne physical therapist certification program
- Successfully implemented a hospital-based newborn screening pilot to screen for Duchenne
- Created an innovative biobank and data registry, facilitating data sharing between patients and researchers
- Provided education, resources and support for thousands of families living with Duchenne, including expertise on clinical trial options and navigating a Duchenne diagnosis.
Prior to founding CureDuchenne, Debra worked in publishing with positions at IDG Communications, Cahners Publishing, Ziff-Davis Publishing, Scholastic Publishing, and more. Miller holds a Bachelor of Arts in Communication Studies from the University of California Los Angeles.
She currently serves on the Alliance for Regenerative Medicine Board, Department of Defense’s Congressionally Directed Medical Research Committee Programmatic Panel, and the Bespoke Gene Therapy Consortium, to name a few. Debra has received numerous awards, including the EY Entrepreneur of The Year™ Award and Orange County Business Journal’s Women in Business Award.
John-Ballantyne John-Ballantyne John is the co-Founder of Aldevron and served as CSO for 23 years before his retirement at the end of 2021. He has vast experience in clinical biologicals manufacturing and delivery methods for nucleic acids.
Pat Furlong Pat Furlong Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Their mission is to end Duchenne. They accelerate research, raise their voices in Washington, demand optimal care for all young men, and educate the global community.
Duchenne is the most common fatal, genetic childhood disorder. It affects 1:4,600 boys worldwide and has no cure.
When doctors diagnosed her two sons, Christopher and Patrick, with Duchenne in 1984, Pat immersed herself in research, working to understand the pathology of the disorder, the extent of research investment and the mechanisms for optimal care.
In 1994, Pat, together with other parents of young men with Duchenne, founded PPMD to change the course of Duchenne and, ultimately, to find a cure. Today, Pat is considered one of the foremost authorities on Duchenne in the world.
Sharon Hesterllee Sharon Hesterllee Dr. Hesterlee is Chief Research Officer of the Muscular Dystrophy Association. She has over 20 years of experience in neuromuscular research in both the nonprofit and industry space. She has previously served as head of research for Parent Project Muscular Dystrophy, the Myotonic Dystrophy Foundation, the Association for Frontotemporal Degeneration and the Muscular Dystrophy Association’s MDA Venture Philanthropy. Dr. Hesterlee has also served as project lead for rare disease gene therapy programs at Pfizer, Inc; as Chief Executive Officer of Lion Therapeutics, a special purpose entity of Askbio, Inc.; and as Executive Vice President and Head of the Neuromuscular Division of Askbio Inc. She has been involved in numerous efforts to remove barriers to therapy development for rare disease and foster interactions between patient advocacy groups and industry. She received her Ph.D. in neuroscience from the University of Arizona in 1999.