SonoThera Presents New Data at the ASGCT Breakthroughs in Muscular Dystrophy Meeting on Ultrasound-Mediated Gene Delivery (UMGD) to Heart and Skeletal Muscle

South San Francisco, CA, November 14, 2024 – SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases through genetic therapy, today announced it will present new data at the American Society of Gene and Cell Therapy (ASGCT) Breakthroughs in Muscular Dystrophy meeting, being held November 19-20, 2024 in Chicago, Illinois.

The presentation focuses on Duchenne Muscular Dystrophy (DMD) and the ability of SonoThera’s ultrasound-mediated nonviral gene (UMGD) therapy platform to potentially provide a safe, redosable, durable and titratable delivery of full-length dystrophin to the heart and skeletal muscles in murine and non-human primate (NHP) models. SonoThera’s novel platform is being developed to non-invasively deliver nucleic acid payloads of diverse formats and sizes, selectively targeting a wide range of organs within the body.

“The recent FDA approval and late stage clinical trials of viral gene therapies for DMD are establishing a new treatment paradigm, however, several significant challenges remain, including failure to improve motor function, inability to deliver full-length dystrophin, viral immunogenicity, inability to redose, safety issues, and high treatment cost,” said Kenneth Greenberg, PhD, CEO of SonoThera. “Ultrasound-mediated gene delivery offers a promising, nonviral, non-invasive approach to address these issues and it’s the focus of the data we’ll be sharing at this important ASCGT meeting.”