SonoThera™ Presents New Data at ESGCT 2025 Demonstrating Expression of Full-Length Human Dystrophin Using Targeted, Ultrasound-Mediated Delivery to Treat Duchenne Muscular Dystrophy

• New data demonstrates the ability of UMD technology to deliver genetic medicines, allowing for full-length human dystrophin protein expression in skeletal, heart and diaphragm muscle tissue in murine and non-human primate (NHP) models.

• Results show high levels of durable human dystrophin protein expression exceeding 50% of endogenous levels have been achieved in NHP.

• Poster presentation spotlights SonoThera’s proprietary, nonviral, non-invasive approach using ultrasound-mediated delivery (UMD).

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases by developing the next generation of genetic medicines, today announced it will present new data at the 32^nd congress of the European Society of Gene and Cell Therapy being held October 7-10 in Seville, Spain.

The poster presentation focuses on treating Duchenne Muscular Dystrophy (DMD) with a full-length human dystrophin payload delivered to skeletal, heart and diaphragm muscles using SonoThera’s proprietary RIPPLE^TM ultrasound-mediated delivery (UMD) technology. The new data demonstrates the ability of RIPPLE^TM to enable broad, highly targeted biodistribution of diverse payload formats without size restriction, to enable genetic medicines that can be re-dosed, and which are safe, well-tolerated, and cost-effective.

“Our new data continues to demonstrate the potential of ultrasound-mediated delivery of genetic medicines and its unique capabilities in treating complex diseases like Duchenne Muscular Dystrophy,” said Kenneth Greenberg, PhD, CEO of SonoThera. “In particular, the ability to deliver targeted, full-length human dystrophin to skeletal, heart and diaphragm muscles in a durable, safe, redosable and noninvasive manner shows how UMD can overcome all of the challenges of current gene therapy delivery options. We look forward to sharing these results at this year’s ESGCT congress.”

Presentation Details:

Poster ID/Presentation: P0266 - Ultrasound-mediated delivery of non-viral full-length dystrophin vector to skeletal, heart and diaphragm muscle tissues in DMD mice and non-human primates.
Category: Cardiovascular and Muscular Diseases
Presenter: Ivan Krivega, PhD, VP of Gene Therapy, SonoThera
Session Date/Time: Thursday, October 9, 2025 1:00pm - 2:30pm CEST (14:00-15:30)
Location: Fibes 2

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.