SonoThera™ to Present New Data at ASGCT 2025 Demonstrating the Potential of Genetic Medicine Using Targeted, Ultrasound-Mediated Delivery in Treating Duchenne Muscular Dystrophy and Hemophilia A

• Oral and poster presentations will highlight SonoThera’s proprietary, nonviral, non-invasive approach using ultrasound-mediated delivery (UMD).

• In the treatment of DMD, data demonstrates the ability of UMD technology to deliver genetic medicines, allowing for full-length dystrophin protein expression in skeletal, cardiac and diaphragm muscles in murine and non-human primate (NHP) models.

• In the treatment of Hemophilia A, preclinical data demonstrates UMD’s ability to non-invasively deliver nonviral, oversized episomal Factor VIII in a durable, titratable and redosable manner.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines, today announced it will present new data at the 28^th annual American Society of Gene and Cell Therapy (ASGCT) meeting being held May 13-17^th in New Orleans, Louisiana.

An oral presentation will focus on the treatment of Hemophilia A while new data in the poster session targets the treatment of Duchenne Muscular Dystrophy (DMD). Both presentations at ASGCT feature SonoThera’s proprietary, nonviral, non-invasive approach using ultrasound-mediated delivery, which enables broad, highly targeted biodistribution of diverse genetic medicine formats without size restrictions, and which enables genetic medicines that can be re-dosed, are safe and well tolerated and are cost-effective.

“We’re pleased to be presenting new data at ASGCT highlighting the potential of ultrasound mediated delivery in solving the key challenges in genetic medicine,” said Kenneth Greenberg, PhD, CEO of SonoThera. “This data continues to demonstrate our unique capabilities, including targeted full-length human dystrophin expression in muscle, and delivering oversized genetic payloads in a durable, safe, redosable and noninvasive manner. We look forward to sharing our latest data at this year’s annual meeting.”

Presentation Details:

Oral Presentation: Development of a Novel, Non-Viral Genetic Therapy for Hemophilia A Utilizing Durable, Redosable, and Titratable Approach of Ultrasound-Mediated Delivering of an Oversized Episomal hFVIII DNA Vector.
Session Title: Liver Targeted Genetic Therapies
Presenter: Ivan Krivega, PhD
Session Date/Time: Wednesday, May 14, 2025, 2:30pm – 2:45pm CT.
Room: 288-290

Poster Presentation: Non-Viral Genetic Medicine for Targeted Delivery of Full-Length Dystrophin to Skeletal, Cardiac, and Diaphragm Muscles in DMD Mouse Models and Non-Human Primates.
Session: Disease Models and Clinical Applications: B8 - Musculo-skeletal Diseases
Presenter: Ivan Krivega, PhD
Session Date/Time: Thursday, May 15, 2025, 5:30pm – 7:00pm CT
Location: Poster Hall, Hall I2

About SonoThera™

SonoThera is a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines. Our nonviral technology is designed to overcome all prevailing limitations of genetic medicine, enabling a pipeline of products which leverages our novel capabilities. Using ultrasound-mediated delivery (UMD), we are developing a proprietary, non-invasive approach which enables broad, highly targeted biodistribution, delivery of diverse genetic payloads without size restriction, in a redosable manner designed to be safe, well-tolerated and cost-effective.

Founded by Drs. Kenneth Greenberg, Michael Davidson, and Steve Feinstein, SonoThera is headquartered in South San Francisco.

Visit us at www.sonothera.com and connect with us on LinkedIn, BlueSky and X/Twitter.