SonoThera to Present at ASN Kidney Week 2024 Demonstrating the Potential of Novel Nonviral Ultrasound-Mediated Gene Therapy Delivery Platform

• Presentations confirm potential of delivering targeted gene therapy using SonoThera’s novel, ultrasound-mediated nonviral gene therapy platform in murine models of X-linked Alport Syndrome (XLAS) and Autosomal Dominant Polycystic Kidney Disease (ADPKD) as well as non-human primates.

• Data demonstrates the ability to deliver oversized nucleic acid payloads non-invasively, to the kidneys using FDA cleared/approved ultrasound components with established safety in humans.

• Results highlight the potential use of targeted nonviral nucleic acid delivery to podocytes and tubule epithelial cells in the kidney, resulting in durable gene expression and the ability to re-dose, and dose titrate with exemplary safety and efficacy.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases through genetic therapy, today announced that it will deliver three presentations at the American Society of Nephrology Kidney Week 2024 annual meeting taking place in San Diego, California, October 23-27, 2024.

Each presentation features SonoThera’s novel, ultrasound-mediated nonviral gene therapy platform, which is being developed to non-invasively deliver nucleic acid payloads of diverse formats and sizes. While the platform is designed to selectively target a wide range of organs within the body, research presented at ASN Kidney Week focuses on its potential in the treatment of X-linked Alport Syndrome and Autosomal Dominant Polycystic Kidney Disease, and its targeted delivery of re-dosable DNA payloads resulting in robust and durable gene expression in podocytes and tubule epithelial cells in the kidneys of murine and NHP models.

“Taken together, these three abstracts demonstrate the potential which targeted, nonviral, ultrasound-mediated gene therapy can offer in diseases where presently, we have some of the greatest challenges in finding effective treatments. We look forward to presenting our results at ASN Kidney Week.” said Kenneth Greenberg, PhD, CEO of SonoThera, who will be presenting the Company’s session focused on nonviral gene therapy for X-linked Alport Syndrome.

Session Details:

Title: Development of a Nonviral Gene Therapy for X-linked Alport Syndrome by Targeted Transcutaneous Ultrasound-Mediated Gene Delivery
Presenter: Kenneth Greenberg, PhD, CEO of SonoThera
Session Category: 1201 Genetic Diseases of the Kidneys: Cystic - Non-Cystic Genetic Kidney Diseases: Disease Genes, Modifiers, and Therapies
Session Date/Time: Thursday, October 24, 5:50pm – 6:00pm, Room 23, Convention Center
Abstract Number: TH-OR90

Title: Development of a Nonviral Genetic Medicine for ADPKD Administered through Targeted Transcutaneous Ultrasound-Mediated Delivery
Presenter: Margot Krivega, PhD
Session Category: 1201 Genetic Diseases of the Kidneys: Cystic - Cystic Kidney Diseases: Clinical Assessment and Therapeutic Directions
Session Date/Time: Thursday, October 24, 10:00am – 12:00pm, Exhibit Hall, Convention Center
Abstract Number: TH-PO431

Title: Kidney-Targeted Delivery of Nonviral Nucleic Acids Using Noninvasive Transcutaneous Ultrasound Enables Safe, Re-dosable, Titratable, and Durable Gene Expression in Mice and Nonhuman Primates
Presenter: Burt Frederich, PhD
Session Category: 1201 Genetic Diseases of the Kidneys: Cystic - Cystic Kidney Diseases: Clinical Assessment and Therapeutic Directions
Session Date/Time: Thursday, October 24, 10:00am – 12:00pm, Exhibit Hall, Convention Center
Abstract Number: TH-PO432

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 an ultrasound-mediated, nonviral gene therapy platform treatments designed to provide patients with the next generation of safe and effective genetic medicines. The platform utilizes sonoporation, a microbubble-mediated biophysical process to non-invasively 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.