SonoThera, a biotechnology company developing ultrasound-mediated nonviral genetic medicines, announced the closing of an oversubscribed $125 million Series B financing round to support the clinical advancement of its lead programs for Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD), while expanding its broader pipeline of targeted and redosable therapies.
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 existing investors ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Inc., Medical Excellence Capital, RA Capital, and Vertex Ventures HC.
As part of the financing, Rajul Jain, M.D., Managing Director at Vida Ventures, and Rakhshita Dhar, M.S., Vice President and Head of Healthcare Venture Investments at Leaps by Bayer, joined SonoThera’s Board of Directors.
The proceeds will be used to move the company’s DMD and ADPKD programs into the clinic, broaden its pipeline across multiple organ systems, and scale its proprietary technology platforms. The funding follows preclinical achievements demonstrating targeted delivery across various tissues and successful delivery of large DNA and RNA payloads.
SonoThera’s platform integrates RIPPLE™, its proprietary ultrasound-mediated delivery technology, with PORE™, a payload engineering platform supporting DNA and RNA therapeutics, gene editing, and gene silencing. The company aims to overcome longstanding limitations of traditional gene therapies, including payload size restrictions, delivery challenges, immune responses, safety concerns, and the inability to administer repeat doses.
Preclinical studies have demonstrated targeted delivery and expression across skeletal muscle, heart, liver, kidney, adipose tissue, and brain. The platform has also shown the ability to deliver large genetic payloads, including full-length dystrophin for DMD and RNA-based payloads for gene silencing applications. SonoThera expects to begin its first clinical trial in DMD in 2027.
The company’s approach utilizes widely available FDA-cleared diagnostic ultrasound systems and commercially available ultrasound contrast agents approved for diagnostic use, enabling an investigational noninvasive outpatient procedure that can be completed in about one hour or less.
Founded in South San Francisco, California, SonoThera is developing a platform intended to enable precise and durable gene expression across multiple tissues while addressing challenges associated with viral delivery approaches. Its pipeline focuses on serious diseases including Duchenne muscular dystrophy and autosomal dominant polycystic kidney disease.
KEY QUOTES:
“We are grateful to have the support of this exceptional group of new and existing investors who share our vision of expanding the potential of genetic medicines. Despite remarkable scientific progress, many diseases remain beyond the reach of today’s genetic medicines. We founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field. We believe our technology has the potential to expand the range of diseases addressable by genetic medicines while enabling more precise, durable, safer, and repeatable therapies for patients.”
Kenneth Greenberg, Ph.D., Co-Founder and Chief Executive Officer, SonoThera
“We at Vida have a long history of investing in and developing genetic medicines, some of which are now used to treat patients across the globe. We believe SonoThera, with its RIPPLE™ delivery and PORE™ payload engineering technologies, has the potential to unlock opportunities in diseases with significant unmet need that have been previously inaccessible to other genetic medicine approaches. We have been deeply impressed by SonoThera’s scientific rigor, pace of execution, and vision, and we are excited to support the company as it advances its first programs into the clinic.”
Rajul Jain, M.D., Managing Director, Vida Ventures
“SonoThera has made impressive progress since its founding and continues to demonstrate the potential of its approach to genetic medicine. The team has built a strong foundation, generated compelling preclinical data, and assembled an outstanding group of investors and partners. We are excited to continue supporting the company as it advances toward the clinic.”
Steven Gillis, Ph.D., Managing Director, ARCH Venture Partners
“At CureDuchenne, we are committed to supporting innovative approaches that have the potential to transform the lives of individuals living with Duchenne muscular dystrophy. SonoThera’s differentiated nonviral platform has the potential to address important challenges in gene delivery and bring treatments to families who have been left waiting. We are proud to support the company’s next phase of growth and clinical development.”
Debra Miller, Founder and CEO, CureDuchenne
