Biotech, Janssen Partner on Ultrasound for Gene Therapy

(Public Domain Pictures, Pixabay)

16 May 2023. A developer of ultrasound bubbles to assist gene therapies is studying use of the technology with Janssen Pharmaceuticals for delivering DNA and RNA treatments. Financial and intellectual property details of the agreement between SonoThera Inc. in South San Francisco and Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson, were not disclosed.

SonoThera is a one year-old biotechnology start-up adapting ultrasound technology to help deliver gene therapies throughout the body. Ultrasound employs high-frequency sound waves to penetrate through the skin, and has become a widely used technique for non-invasively diagnosing disorders such as heart disease and picturing a fetus in the womb, in real time and without radiation.

SonoThera seeks to extend ultrasound use to gene therapies, which the company says are limited by current delivery methods. In many cases today, notes SonoThera, gene therapies are delivered with benign viruses that are often complex and expensive, have a small payload capacity and limited cell or tissue targets, and while harmless to many people may still cause an immune reaction in some patients. Non-viral gene therapy delivery methods, adds the company, are so far inefficient and time-limited in effectiveness, as well as poorly targeted and toxic in some cases.

Larger and wider range of payloads

SonoThera says its technology uses ultrasound to create microscale bubbles in a process called sonoporation that increases the uptake of gene therapies through cell membranes. The company says its process can deliver larger and a wider range of genetic payloads, and to more organs in the body than most other gene therapy delivery methods. In addition, the demonstrated safety of ultrasound for diagnostics increases the number of patients who can receive gene therapies. In June 2020, Science & Enterprise reported on an academic lab conducting preclinical research that demonstrates ultrasound assisting genetic immunotherapy deliveries in lab mice induced with solid-tumor cancers.

The agreement with Janssen Pharmaceuticals calls for the companies to jointly research ultrasound to help deliver nucleic acids such as DNA and RNA payloads to organs other than the liver. “We remain deeply committed,” says SonoThera co-founder and CEO Kenneth Greenberg in a company statement released through Cision, “to developing genetic medicines targeting organ systems which pose significant delivery challenges using existing gene therapy modalities and look forward to further accelerating key programs within our non-viral gene therapy platform development through this collaboration.”

While SonoThera is only about a year-old, the company already has a firm relationship with Janssen and its Johnson & Johnson parent. Before founding SonoThera, Feinberg was senior director for external innovation at Janssen R&D. Also, in December 2022, SonoThera raised nearly $61 million in its first venture round, with Johnson & Johnson Innovation taking part in the financing. Plus, SonoThera says Johnson & Johnson Innovation helped arrange for the new collaboration with Janssen Pharma.

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Disclosure: The author owns shares in Johnson & Johnson.

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