SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by developing the next generation of genetic medicines, announced on January 8, 2026, that its proprietary RIPPLE™ technology had detected strong and persistent full-length human dystrophin protein expression in the skeletal muscles of non-human primates, reaching up to 290% of normal levels.
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To learn more about SonoThera’s RIPPLE technology firsthand, we contacted Elizabeth Harness from the Corporate Communications department and shared our questions on behalf of our followers. Elizabeth thoughtfully and comprehensively provided details about Ultrasound-Mediated Delivery (UMD) technology, which is also planned for use in Duchenne muscular dystrophy.
SonoThera, a South San Francisco–based genetic medicine company, announced the initiation of a $125 million Series B financing round at the 44th J.P. Morgan Healthcare Conference. The capital raised will be used to advance first-in-human clinical studies for three lead programs targeting Duchenne muscular dystrophy (DMD), autosomal dominant polycystic kidney disease (ADPKD), and X-linked Alport syndrome (XLAS). These programs will leverage SonoThera’s proprietary RIPPLE platform, a nonviral, ultrasound-mediated delivery technology designed to overcome the limitations of viral vectors by enabling targeted, size-independent payload delivery and supporting scalable and repeatable gene therapy development.
SonoThera’s nonviral platform: RIPPLE
SonoThera’s approach to genetic delivery directly addresses one of the most enduring challenges in gene therapy: the inherent constraints of viral vectors. While adeno-associated virus (AAV) remains the prevailing delivery modality, it is limited by a relatively small cargo capacity of approximately 4.7 kb, potential immunogenic responses, and difficulties associated with repeat dosing due to the development of neutralizing antibodies. These drawbacks are especially problematic for conditions that require delivery of large genetic sequences, with Duchenne muscular dystrophy standing as a clear example.
The company’s RIPPLE platform, which leverages ultrasound-mediated delivery (UMD), is designed to bypass these barriers. By combining focused ultrasound with microbubble activation, the technology temporarily increases cell membrane permeability, enabling targeted uptake of genetic material independent of payload size. Importantly, the platform is positioned as being redosable, long-lasting, and suitable for full-length gene delivery—opening therapeutic possibilities for diseases that have been difficult to address using conventional vector-based systems.
From a broader platform standpoint, RIPPLE represents more than an incremental advancement in delivery technology. Industry observers suggest it could help define a new class of nonviral, device-assisted gene therapies, particularly as regulatory scrutiny of viral vectors intensifies in response to ongoing manufacturing and safety challenges.
Datas of full-length human dystrophin raises expectations
The selection of DMD as a priority indication indicates that SonoThera aims to move beyond the limitations of existing treatment paradigms. Currently approved AAV-based therapies for DMD, including Sarepta Therapeutics’ Elevidys, depend on micro-dystrophin constructs because of the strict payload constraints imposed by AAV vectors. While these shortened genes can slow disease progression, they are not curative and do not fully restore muscle function.
By comparison, SonoThera has reported expression of full-length human dystrophin in skeletal muscle of non-human primates at levels reaching up to 290% of normal, along with demonstrated recovery of muscle strength in rodent models. Should these results translate even partially to clinical settings, they could represent a transformative advance in DMD therapy—potentially achieving levels of therapeutic benefit approaching those observed in Becker muscular dystrophy or in asymptomatic dystrophin mutation carriers.
Questions We Asked SonoThera and the Answers We Received
In SonoThera’s press release, company mentioned conducting a study on non-human primates. In this study, which genetic medicine did company use to deliver to organs using UMD technology?
Genetic medicine is a broad category that includes many types of gene therapies. SonoThera is a gene therapy company; however, unlike currently approved DMD gene therapies, we do not use viral delivery vehicles such as AAV.
Instead, SonoThera has exclusively developed a novel, non-viral delivery platform called RIPPLE™. RIPPLE™ was designed specifically to overcome the safety, efficacy, and cost/manufacturing limitations of existing gene delivery approaches.
Our proprietary approach enables safe, efficient, and uniform biodistribution of genetic payloads throughout the target organ. In Duchenne muscular dystrophy, our focus is on delivery of a DNA molecule encoding full-length dystrophin to skeletal muscle, heart, and diaphragm, while minimizing the risk of off-target delivery. Because RIPPLE™ is non-viral, it is immune-stealth and fundamentally different from gene delivery platforms used in approved DMD therapies. The technology is also designed to be durable and re-dosable.
RIPPLE™ uses commercially available ultrasound systems, probes, and microbubbles to deliver genetic payloads non-invasively. The microbubbles and genetic payload are administered together intravenously and circulate throughout the body. Ultrasound energy is then applied specifically to the target tissue, causing the microbubbles to collapse and create transient pores. This allows the genetic payload to exit the blood vessels, enter muscle cells, and subsequently reach the nucleus, where gene expression can occur.
To confirm, we ask: Is SonoThera using a genetic medicine developed in-house? That is, not a gene therapy that is already on the market or undergoing clinical trials!
SonoThera has developed a novel, non-viral delivery platform called RIPPLE™ which uses commercially available ultrasound systems, probes, and microbubbles to deliver genetic payloads non-invasively. In terms of genetic payloads or “medicines”, our approach allows us to use a variety of different kinds of genetic payloads without size limitation. Most importantly, we do not use viral delivery vehicles such as AAV which is why our approach is non-viral.
In terms of the payload we are using for the DMD study, we are using DNA for cell replacement. This payload and its elements are proprietary to SonoThera.
Which tests were used to determine if full-length dystrophin production was achieved? Did SonoThera measure it via muscle biopsy?
Yes. Full-length dystrophin expression was evaluated using muscle biopsies collected following treatment. These samples were analyzed using controlled laboratory methods, including Western Blot to confirm the presence of full-length dystrophin protein, as well as immunohistochemistry to assess muscle fiber biodistribution and appropriate subcellular localization over time.
Did SonoThera have the opportunity to see the Creatine Kinase, AST, and ALT results? How much did these values decrease?
We have evaluated AST, ALT, and additional markers of immune response, and to date we have not observed safety issues following either single or repeat treatment in both small and large animal models.
We are currently in the process of evaluating reductions in creatine kinase (CK) following treatment of Duchenne disease models with full-length dystrophin.
When does SonoThera plan to begin studies on humans?
We are preparing for clinical development and are targeting initiation of our first human clinical trial in the first half of 2027, pending regulatory review.
The road to clinic trials
Outside the clinical setting, evolving regulatory dynamics may further strengthen SonoThera’s position. Both the FDA and EMA have begun placing greater emphasis on advances in delivery technologies, particularly following setbacks in AAV-based programs and reports of patient fatalities in high-dose systemic studies. Demonstrating a safe, nonviral gene delivery approach that allows for repeat dosing could have far-reaching implications, shaping regulatory expectations as well as influencing future academic research and commercial platform strategies across the field.
Should SonoThera succeed in realizing even a portion of its technological potential, the RIPPLE platform may transition from an emerging innovation to a foundational framework for the next generation of nonviral gene therapies.
Important Notice from SonoThera for Patients and Families:
Please be aware that SonoThera is not able to accept or review personal medical information under any circumstances. From time to time, we receive messages from patients, families, or caregivers seeking to share medical details in the hope of participating in a future clinical trial. However, applicable laws and regulatory requirements strictly prohibit us from receiving, storing, or evaluating such information.
When SonoThera initiates its first human clinical trial, the announcement will be made publicly through an official press release and the company’s social media channels, along with a direct link to the study listing on an approved clinical trials database. We strongly encourage readers to rely only on these official communications and to follow updates via our website, LinkedIn, BlueSky, and Twitter/X accounts. >>> LinkedIn | BlueSky | Twitter/X
