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 32nd 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 RIPPLETM ultrasound-mediated delivery (UMD) technology.
Ultrasound-Mediated Delivery
The new data demonstrates the ability of RIPPLETM 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. – What is Ultrasound-Mediated Delivery Method? –
Full-Length Human Dystrophin
- 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).
“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.”
Read More: Clinical Trials for Duchenne (List of All Researches)
