- Robust full-length dystrophin expression with phenotypic correction in muscle for Duchenne muscular dystrophy (DMD)
- Targeted siRNA knockdown across multiple extrahepatic tissues, including muscle, kidney, brain, and adipose
- Redosable delivery of oversized Factor VIII (FVIII) for hemophilia A, achieving up to 65% of normal levels
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--#ASGCT26--SonoThera, a biotechnology company developing next-generation genetic medicines to address the root causes of disease, today announced upcoming presentations at the 2026 American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, May 11-15 in Boston, Massachusetts.
Data to be presented will highlight the breadth and versatility of SonoThera’s proprietary RIPPLE™ platform, a non-viral, immune-stealth ultrasound-mediated gene delivery system designed to enable safe, redosable genetic medicines across multiple tissues and disease areas.
“These data highlight the potential of our RIPPLE platform to enable targeted, redosable delivery of genetic medicines across multiple tissues and to build a broad pipeline across multiple indications,” said Kenneth Greenberg, Ph.D., Chief Executive Officer of SonoThera.
In oral presentations, SonoThera will share preclinical data demonstrating robust delivery and activity of therapeutic payloads across muscle, kidney, adipose, and brain. The data will include full-length dystrophin expression and phenotypic correction in models of Duchenne muscular dystrophy (DMD), as well as efficient and targeted siRNA knockdown in extrahepatic tissues. These findings highlight the potential of RIPPLE™ to address key limitations of viral delivery approaches, including payload size constraints, immunogenicity, and lack of redosability.
In addition, a poster presentation will feature data on non-viral delivery of oversized Factor VIII (FVIII) for hemophilia A in non-human primates, achieving sustained expression of up to 65% of physiologic FVIII levels for 60 days. These results demonstrate the platform’s ability to enable delivery and expression of large therapeutic proteins that have historically been challenging with conventional approaches.
Details for the presentations are as follows:
Oral Presentation Title: Robust siRNA knock-down in the muscle, kidney, adipose and brain using RIPPLE™ non-viral noninvasive immune-stealth delivery platform
Oral Presentation #: 59
Session: Emerging non viral approaches for tissue and cell specific delivery
Session Date and Time: Tuesday, May 12, 2026, 11:45 AM ET
Oral Presentation Title: Amelioration of DMD disease phenotype through full-length dystrophin protein expression using SonoThera's RIPPLE™, a non-viral, non-invasive, immune stealth gene delivery platform
Oral Presentation #: 209
Session: Translational advances in genetic and cellular therapies for muscle and skeletal disorders
Session Date and Time: Wednesday, May 13, 2026, 10:45 AM ET
Poster Title: Non-Viral, Safe, Redosable Oversized FVIII Expression for Hemophilia A Using SonoThera’s RIPPLE™ Platform
Poster # 3513
About SonoThera™
SonoThera is a biotechnology company focused on treating the root causes of disease through next-generation genetic medicines. The company’s proprietary RIPPLE™ platform is a non-viral, immune-stealth gene delivery system designed for targeted, redosable administration of diverse genetic payloads across multiple tissues, enabling a broad pipeline of genetic medicines across multiple indications. Leveraging ultrasound-mediated delivery (UMD), the platform enables broad biodistribution, supports delivery of large genetic constructs without size constraints, and is designed for repeat dosing with a favorable safety profile. SonoThera is headquartered in South San Francisco. For more information, visit www.sonothera.com.
Contacts
Investor Contact
investors@sonothera.com
Media Contact
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
