• New data demonstrates SonoThera’s novel, ultrasound-mediated gene delivery (UMGD) platform’s ability to noninvasively provide safe, effective gene therapy delivery to liver.

• Presentation focuses on the platform’s potential for treating hematological diseases such as Hemophilia A and B, and von Willebrand disease.

SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases through genetic therapy, today announced it will present new data on its novel gene delivery platform and therapeutic programs at the 66^th American Society of Hematology (ASH) Annual Meeting and Exposition, being held December 7-10^th in San Diego, California.

The preclinical data highlights the ability of ultrasound-mediated gene delivery (UMGD) to deliver nonviral genetic payloads in a durable, titratable, redosable manner while achieving therapeutic FVIII and FIX gene expression levels in murine models. SonoThera’s novel UMGD platform is being developed to noninvasively deliver nucleic acid payloads of diverse formats and sizes safely, selectively targeting a wide range of organs and tissues within the body.

“A major challenge to creating safe and effective genetic treatments for hematological diseases such as Hemophilia A and B, and von Willebrand disease remains the ability to efficiently deliver oversized, nonviral DNA payloads to the liver in a safe and redosable manner,” said Kenneth Greenberg, PhD, CEO of SonoThera. “Ultrasound-mediated gene delivery has the ability to address these key issues, and we look forward to presenting our latest data demonstrating its potential.”

Presentation Details:

Publication Number: 2232

Title: Targeted Non-Viral Gene Delivery through Transcutaneous Ultrasound Enables Robust, Redosable, Titratable, and Durable FVIII and FIX Gene Expression in the Liver in Murine Models.

Session: 803. Emerging Tools, Techniques, and Artificial Intelligence in Hematology: Poster I

Date: Saturday, December 7, 2024

Time: 5:30 pm – 7:30 pm PST

Location: San Diego Convention Center, Halls G-H

About SonoThera

Founded by Drs. Kenneth Greenberg, Michael Davidson, and Steve Feinstein, SonoThera, Inc. is a biotechnology company dedicated to treating the root cause of human disease through genetic therapy. SonoThera is developing a nonviral, ultrasound-mediated gene delivery platform designed to provide patients with the next generation of safe and effective genetic medicines. The novel platform utilizes sonoporation, a microbubble-mediated biophysical process to noninvasively deliver genetic payloads selectively targeting a wide range of organs within the body. SonoThera is headquartered in South San Francisco, California. Visit www.sonothera.com to learn more.

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The preclinical data highlights the ability of ultrasound-mediated gene delivery (UMGD) to deliver nonviral genetic payloads in a durable, titratable, redosable manner while achieving therapeutic FVIII and FIX gene expression levels in murine models.