SonoThera will present new data at the 2025 MDA Conference demonstrating the potential of genetic medicine using targeted, Ultrasound-Mediated Delivery (UMD) in treating Duchenne Muscular Dystrophy.
- New preclinical data demonstrates the ability of SonoThera’s novel, nonviral UMD technology to deliver genetic medicines, allowing for full-length dystrophin protein expression in skeletal, cardiac and diaphragm muscles.
- SonoThera’s non-invasive approach is being developed to enable broad, highly targeted biodistribution of diverse genetic medicines without size restriction in a safe, redosable manner.
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 annual Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 16-19th in Dallas, Texas.
Ultrasound-Mediated Delivery (UMD)
SonoThera is developing the world’s first ultrasound-mediated nonviral gene delivery platform, which is designed to overcome the limitations of other gene therapy approaches.
The preclinical data highlights the ability of ultrasound-mediated delivery (UMD) to express nonviral genetic constructs in a redosable, durable manner, allowing full-length dystrophin expression in skeletal, cardiac and diaphragm muscles in murine models. SonoThera is developing a proprietary, nonviral, non-invasive approach using ultrasound-mediated delivery, which enables broad, highly targeted biodistribution of diverse genetic medicines without size restriction in a redosable manner, designed to be safe, well-tolerated and cost-effective.
“We are focused on the significant challenges that remain for the treatment of DMD which include the inability to deliver full-length dystrophin, viral vector immunogenicity, failure to improve motor function, inability to redose, safety issues, and of course, the high cost of treatment,” said Kenneth Greenberg, PhD, CEO of SonoThera. “We believe UMD can solve these issues and do so in a nonviral, non-invasive way. We look forward to sharing our latest data at the upcoming MDA conference.”