SonoThera's Ultrasound Gene Therapy Achieves 65% Normal Factor VIII Levels in Hemophilia A Primate Study
Key Insights
SonoThera (search)'s RIPPLE (search) ultrasound-mediated delivery technology demonstrated up to 65% of normal Factor VIII (search) protein expression in non-human primate models for hemophilia A (search) treatment.
The nonviral gene therapy approach enables targeted liver delivery without size restrictions and allows for re-dosing, addressing limitations of current approved therapies.
Study results were presented at the 67th American Society of Hematology Annual Meeting, showing durable protein expression using DNA expression vectors delivered via ultrasound.
SonoThera (search) presented breakthrough data at the 67th American Society of Hematology (ASH) Annual Meeting demonstrating that its proprietary ultrasound-mediated gene delivery technology achieved up to 65% of normal Factor VIII (search) protein expression in non-human primate models for treating hemophilia A (search).
The biotechnology company's oral presentation focused on the safe delivery of DNA expression vectors to the liver using its RIPPLE (search) ultrasound mediated delivery (UMD) technology. The study results showed durable protein expression of up to 65% of normal circulating FVIII levels in the blood of non-human primates.
Novel Ultrasound-Mediated Delivery Platform
RIPPLE (search) technology represents a nonviral, non-invasive approach designed to enable broad, highly targeted biodistribution of diverse payload formats without size restriction. This capability allows for genetic medicines that can be re-dosed and are designed to be safe, well-tolerated, and cost-effective.
"These new, positive results only further build upon our promising data previously presented at ASH," said Kenneth Greenberg, PhD, SonoThera (search) CEO. "We continue to demonstrate UMD's potential to safely, efficiently deliver an oversized payload to the liver resulting in high levels of functional FVIII expression."
Addressing Current Treatment Limitations
According to Greenberg, current approved therapies for Hemophilia A (search) face significant challenges including variable expression, unfavorable durability, and immunogenicity. The company's UMD technology has demonstrated its ability to address these therapeutic limitations in preclinical studies.
The ultrasound-mediated delivery approach uses targeted payload delivery to the liver, enabling the delivery of genetic payloads without the size restrictions that limit other gene therapy approaches. This nonviral technology is designed to overcome prevailing limitations of genetic medicine.
Clinical Development Strategy
SonoThera (search)'s technology platform is being developed to treat the root cause of human diseases through next-generation genetic medicines. The company's proprietary approach enables broad, highly targeted biodistribution and delivery of diverse genetic payloads in a redosable manner.
The research was presented during the Gene Therapies: Technological Developments in Gene Therapy session at ASH 2025, with the presentation titled "Non-viral ultrasound-mediated delivery of an episomal FVIII expression vector enables durable FVIII protein production at therapeutic levels in non-human primates."
Ivan Krivega, PhD, VP of Gene Therapy at SonoThera (search), delivered the presentation on December 8th at the Orange County Convention Center in Orlando, Florida.