SonoThera has secured $125 million in an oversubscribed Series B financing round to support the clinical development of its ultrasound-mediated, nonviral genetic medicine platform. The South San Francisco-based biotechnology company said the funding will help advance its lead programs in Duchenne muscular dystrophy and autosomal dominant polycystic kidney disease toward human studies. The round also strengthens investor backing for SonoThera’s broader ambition to develop targeted and redosable genetic medicines for serious diseases.
Financing Round Draws Major Healthcare Investors
The financing was led by Vida Ventures and included participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital, and several existing investors. Returning backers included ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Medical Excellence Capital, RA Capital, and Vertex Ventures HC. As part of the transaction, Vida Ventures Managing Director Rajul Jain and Leaps by Bayer’s Rakhshita Dhar joined SonoThera’s board of directors.
Funding to Advance Lead Programs
SonoThera plans to use the proceeds to move its lead programs in Duchenne muscular dystrophy, known as DMD, and autosomal dominant polycystic kidney disease, known as ADPKD, into clinical development. The company will also use the capital to broaden its pipeline across multiple organ systems and further scale its core platform technologies. SonoThera said the financing follows preclinical progress showing targeted delivery across several tissues and the ability to deliver both large DNA and RNA payloads.
Platform Designed to Address Gene Therapy Limits
The company’s approach combines RIPPLE, its proprietary ultrasound-mediated delivery technology, with PORE, a payload engineering platform designed to support DNA therapeutics, RNA therapeutics, gene editing, and gene silencing. SonoThera says the platform is being developed to overcome several barriers associated with existing genetic medicine approaches, including limited tissue delivery, payload size restrictions, immune responses, safety concerns, and difficulties with redosing. By avoiding viral delivery methods, the company aims to create therapies that can be administered repeatedly and directed more precisely to target tissues.
Preclinical Data Supports Broader Development
SonoThera has reported targeted delivery and expression in preclinical studies across skeletal muscle, heart, liver, kidney, adipose tissue, and brain. The company has also demonstrated delivery of large genetic payloads, including full-length dystrophin for DMD, as well as RNA-based payloads for gene silencing applications. SonoThera expects to begin its first clinical trial in DMD in 2027, marking a key step in testing whether its nonviral delivery approach can translate from preclinical research to patient treatment.
Investor Support Reflects Platform Potential
Kenneth Greenberg, SonoThera’s co-founder and chief executive officer, said the company was created to expand the potential reach of genetic medicines beyond current limitations. He noted that many diseases remain difficult to address with today’s available technologies, despite major advances across the field. Investors including Vida Ventures, ARCH Venture Partners, and CureDuchenne highlighted the company’s scientific progress, differentiated delivery strategy, and potential relevance for diseases with substantial unmet medical need.
Noninvasive Delivery Model
SonoThera’s platform is designed to use widely available diagnostic ultrasound systems cleared by the U.S. Food and Drug Administration, along with commercially available ultrasound contrast agents approved for diagnostic use. The company says this could support an investigational outpatient procedure that is noninvasive and may be completed in about one hour or less. If validated clinically, that model could offer a practical alternative to more complex genetic medicine delivery approaches.
The $125 million Series B financing gives SonoThera significant resources to push its lead genetic medicine programs toward the clinic while continuing to expand its technology platform. With DMD and ADPKD as its initial focus areas, the company is positioning ultrasound-mediated nonviral delivery as a potential solution to some of the most persistent challenges in genetic medicine. The upcoming clinical transition, beginning with a planned DMD trial in 2027, will be an important test of whether SonoThera’s platform can deliver on its promise for patients.