SonoThera's Ultrasound Platform Aims to Reshape Gene Therapy

SOUTH SAN FRANCISCO, CA – May 05, 2026 – By Matthew Richardson

In the rapidly advancing world of genetic medicine, the holy grail has long been a delivery method that is safe, precise, and can be administered more than once. South San Francisco-based SonoThera is poised to argue it has found just that, announcing it will present groundbreaking preclinical data for its proprietary RIPPLE™ platform at the upcoming American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting in Boston. The technology, a non-viral gene delivery system using ultrasound, promises to sidestep the critical limitations that have constrained the revolutionary potential of gene therapy for years.

SonoThera’s upcoming presentations will showcase the platform's versatility, with data demonstrating robust gene delivery and expression in a range of tissues—from muscle and kidney to the brain. The company is tackling some of the most challenging genetic disorders, including Duchenne muscular dystrophy (DMD) and hemophilia A, with a novel approach that could fundamentally alter treatment paradigms.

Overcoming the Viral Hurdle

For decades, the workhorse of gene therapy has been the adeno-associated virus (AAV), a re-engineered virus used as a vector to deliver therapeutic genes into cells. While AAVs have led to landmark approvals, they are fraught with inherent challenges. Many patients have pre-existing antibodies that render the therapy ineffective. The body’s immune response to the viral vector typically prevents redosing, making most AAV therapies a one-shot-only treatment. Furthermore, AAVs have a limited packaging capacity, often less than 5 kilobases, which is too small to carry the full-length version of many crucial genes.

SonoThera’s RIPPLE™ platform is designed to bypass these obstacles entirely. The system employs a non-viral, two-part process known as ultrasound-mediated delivery (UMD). First, microbubbles are injected into the bloodstream alongside the desired genetic payload. Then, using an FDA-cleared ultrasound device, acoustic energy is precisely focused on the target tissue. This energy causes the microbubbles to oscillate and create transient, microscopic pores in cell membranes—a process called sonoporation—allowing the genetic material to enter the cells directly.

This method carries significant advantages. By avoiding viruses, it is “immune-stealth,” drastically reducing the risk of an immune response and opening the door for repeat administrations. This redosability is critical for managing chronic conditions or for patients who may need dose adjustments over time. The platform also liberates developers from the size constraints of viral vectors, enabling the delivery of large and complex genetic payloads.

New Hope for Debilitating Diseases

The true test of any platform technology lies in its real-world application. SonoThera is bringing forward compelling preclinical data in two diseases with high unmet needs, demonstrating the tangible benefits of its approach.

For Duchenne muscular dystrophy, a devastating muscle-wasting disease, the company will present data showing the delivery of the full-length dystrophin gene. Current approved gene therapies for DMD use a truncated “micro-dystrophin” due to the AAV vector’s size limitations. While beneficial, it is not the complete protein. SonoThera’s data, generated in non-human primate models, reportedly shows robust expression of full-length dystrophin—exceeding 50% of normal levels—in key muscles, including the heart and diaphragm, which are critical for survival in DMD patients.

Similarly, for hemophilia A, a bleeding disorder caused by a lack of Factor VIII (FVIII) protein, SonoThera is tackling the dual challenges of gene size and treatment durability. The company will present poster data on the non-viral delivery of an oversized FVIII gene in non-human primates. The results show sustained expression of up to 65% of physiologic FVIII levels for 60 days. This is particularly significant as some AAV-based hemophilia therapies have faced questions about long-term durability, with protein levels declining over time.

“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 a recent statement.

A Platform Poised for Clinical Translation

The promise of a versatile, redosable gene therapy platform has not gone unnoticed by the investment community. SonoThera has secured significant financial backing, including a $60.75 million Series A round in late 2022 led by ARCH Venture Partners and a more recent $125 million Series B round. The roster of investors includes a who's who of venture capital and pharmaceutical giants, such as Eli Lilly & Company, Johnson & Johnson Innovation, and Vertex Ventures, signaling strong confidence in the technology’s disruptive potential.

This capital is fueling the company’s push toward the clinic. The explicit goal of the latest funding round is to advance multiple genetic medicine programs into first-in-human studies. The breadth of the RIPPLE™ platform, demonstrated by its ability to target not only muscle and liver but also kidney, adipose, and brain tissue, positions SonoThera to pursue a wide-ranging therapeutic pipeline rather than being limited to a single disease or organ system.

As SonoThera prepares to step into the spotlight at ASGCT, the broader biotech community will be watching closely. The full data presented at the meeting will offer a deeper look into the efficacy and safety profile of the RIPPLE™ platform. If the technology lives up to its promise, it could represent a pivotal shift in genetic medicine, offering a safer, more versatile, and durable treatment option for countless patients who have been waiting for a breakthrough.