San Francisco — SonoThera said it has closed an oversubscribed $125 million Series B financing round to advance its ultrasound-mediated nonviral genetic medicine programs into clinical development.
The financing was led by Vida Ventures, with participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital and existing investors.
SonoThera said proceeds will support clinical advancement of its lead programs in Duchenne muscular dystrophy and autosomal dominant polycystic kidney disease. The funding will also help expand the company’s pipeline of targeted, redosable genetic medicines across multiple organ systems and further scale its proprietary platform technologies.
Existing investors in the round included ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson through Johnson & Johnson Innovation – JJDC, Inc., Medical Excellence Capital, RA Capital and Vertex Ventures HC.
In connection with the financing, Rajul Jain, M.D., Managing Director at Vida Ventures, and Rakhshita Dhar, M.S., Vice President & Head, Healthcare Venture Investments at Leaps by Bayer, joined SonoThera’s board of directors.
SonoThera’s platform combines RIPPLE, its proprietary ultrasound-mediated delivery technology, with PORE, a payload engineering platform that supports DNA and RNA therapeutics, gene editing and gene silencing approaches.
The company said the technologies are designed to develop targeted, redosable genetic medicines that address key limitations of conventional gene therapies, including delivery challenges, payload size limits, immune responses, safety issues and the inability to redose.
“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,” said Kenneth Greenberg, Ph.D., Co-Founder and Chief Executive Officer, SonoThera. “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.”
Rajul Jain, M.D., Managing Director at Vida Ventures, said SonoThera’s platform could open new opportunities in diseases that have been difficult to address with existing genetic medicine approaches.
“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,” Jain said. “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.”
SonoThera said it has demonstrated targeted delivery and expression across multiple tissues, including skeletal muscle, heart, liver, kidney, adipose and brain. The company has also shown delivery of large genetic payloads, including full-length dystrophin for Duchenne muscular dystrophy, and RNA-based payloads for gene silencing applications in preclinical studies.
The company expects to begin its first clinical trial in Duchenne muscular dystrophy in 2027.
“SonoThera has made impressive progress since its founding and continues to demonstrate the potential of its approach to genetic medicine,” said Steven Gillis, Ph.D., Managing Director at ARCH Venture Partners. “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.”
Debra Miller, Founder and CEO of CureDuchenne, said SonoThera’s nonviral platform has the potential to address gene delivery challenges for people with Duchenne muscular dystrophy.
“At CureDuchenne, we are committed to supporting innovative approaches that have the potential to transform the lives of individuals living with Duchenne muscular dystrophy,” Miller said. “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.”
SonoThera said its approach uses widely available, FDA-cleared diagnostic ultrasound systems and commercial ultrasound contrast agents approved for diagnostic uses, enabling an investigational, noninvasive outpatient procedure that can be performed in about one hour or less.
