• New data involves nonviral, targeted payload delivery to the liver using ultrasound mediated delivery (UMD), resulting in up to 65% of normal Factor VIII protein expression in non-human primate (NHP) models.
• Oral presentation highlights RIPPLE^™, SonoThera’s proprietary, nonviral, non-invasive ultrasound-mediated gene delivery technology.

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 67^th American Society of Hematology (ASH) Annual Meeting and Exposition, being held December 6-9^th in Orlando, Florida.

SonoThera’s oral presentation focuses on the safe delivery of DNA expression vectors to the liver in non-human primate (NHP) models using its proprietary RIPPLE^™ ultrasound mediated delivery technology. Study results demonstrated durable protein expression of up to 65% of normal circulating FVIII levels in the blood. RIPPLE^™ is being developed to enable broad, highly targeted biodistribution of diverse payload formats without size restriction, allowing for genetic medicines that can be re-dosed, and which are 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 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. Where current approved therapies for Hemophilia A are limited by variable expression, unfavorable durability, and immunogenicity, UMD has, thus far, shown its ability to address these challenges, and we look forward to sharing this data.”

Session Details:

Session: 801. Gene Therapies: Technological Developments in Gene Therapy

Title: Non-viral ultrasound-mediated delivery of an episomal FVIII expression vector enables durable FVIII protein production at therapeutic levels in non-human primates

Abstract Number: 1066 Presentation ID: abs25-13006

Date & Time: Monday, December 8^th 5:15-5:30 pm ET

Location: Orange County Convention Center, Sunburst Room (W340)

Presenter: Ivan Krivega, PhD, VP of Gene Therapy, SonoThera

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.

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Where current approved therapies for Hemophilia A are limited by variable expression, unfavorable durability, and immunogenicity, UMD has, thus far, shown its ability to address these challenges, and we look forward to sharing this data.