• Therapeutic expression with durability past 6 months modeling hemophilia B treatment in non-human primate model
• Safe and redosable expression of DNA into the liver of pig and non-human primate models.
• Non-viral delivery and expression into genetic pig models of tyrosinemia and phenylketonuria showing ability to ameliorate relevant biomarkers.

CAMBRIDGE, Mass., May 11, 2026 /PRNewswire/ — HydroGene Therapeutics is a biotechnology company creating accessible gene therapies for all liver diseases. HydroGene is proud to announce our upcoming presentations at the 2026 American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, May 11-15 in Boston, Massachusetts.

The ASGCT Meeting will be the debut of HydroGene’s platform data as the company demonstrates the significant advances in gene delivery into the liver non-human primates (NHP) as well as large animal models of genetic disease. HydroGene believes the achievements represent a significant advance for the field of non-viral gene therapy, which previously suffered significant toxicities, and low-level expression with limited duration in large animal models. In addition, the technology solves current limitations around adeno-associated virus (AAV) therapies, including high manufacturing costs and complexity, safety issues, prolonged need for immunosuppression, and immunity restricting initial eligibility and preventing redosing.

HydroGene’s core technology efficiently scales hydrodynamic injection into large animal models. Hydrodynamic injection is a fluid pressure-based gene delivery procedure, where naked DNA enters inside cells within seconds in an organ-specific manner. HydroGene’s unique approach leverages the biliary system of the liver to overcome systemic administration concerns. Access to the biliary system is widely available today through a simple non-surgical procedure in the form of endoscopic retrograde cholangiopancreatography (ERCP), which is performed in hospitals across the world.

“Previous efforts had struggled to scale hydrodynamic injection from mouse to large animal models,” said Robert Kruse, MD, PhD, co-founder and CEO of HydroGene. “Our earlier work at Johns Hopkins had demonstrated the feasibility of safely injecting DNA into human-sized pig models. We’re now excited to extend the platform into the non-human primate model, the gold standard for gene therapy, as well as investigate treating large animal models of genetic disease.”

In HydroGene’s oral presentation, HydroGene will share the ability to dose DNA into NHP liver, such that the expression levels are similar to the FDA approved AAV-based hemophilia B products. Stability and expression past six months have also been achieved, as well as reproducibility of the gene delivery method across multiple different individual NHPs. HydroGene will share additional data around the feasibility of redosing genetic medicine into NHP as well as safety data showing that the procedure is well tolerated. Together, these accomplishments are the central tenets for the ideal genetic medicine that current AAV viral vector platforms are unable to achieve.

In addition, HydroGene will present two poster presentations detailing the company’s efforts and pushing forward treatments and to specific rare diseases of Wilson disease, phenylketonuria, and hereditary tyrosinemia. These posters illustrate the ability for hydrodynamic delivering pigs to mediate high transfected areas, as well as demonstrate for the first time the ability of a non-viral gene therapy to mediate correction of relevant biomarkers in an authentic genetic model of those diseases. This achievement sets the stage for translation into human patients with these disorders after further optimization. A final poster presentation will highlight a novel platform that HydroGene has developed for site-specific insertion of DNA into the liver through hydrodynamic delivery of naked DNA and Cas9 ribonucleoprotein. The platform yielded therapeutic expression for hemophilia and tyrosinemia models in mice, respectively.

With these achievements shared at ASGCT 2026, HydroGene is preparing for a first-in-human study in 2027 to demonstrate non-viral DNA delivery into the liver of hemophilia patients. “We have received positive feedback on our approach across several genetic diseases to address the unmet clinical need,” said Vivek Kumbhari, MBChB, PhD, co-founder of HydroGene and Professor of Medicine at Mayo Clinic. “Through a well-tolerated outpatient endoscopic procedure, we believe we have a safe approach for genetic medicine that avoids the toxicities and concerns of viral approaches.” Founded in 2022 and emerging from stealth, HydroGene is supported by Curie.Bio and Liquid2 Ventures, as well as research collaborations and grants with Johns Hopkins and Mayo Clinic.

Details for the ASGCT 2026 presentations are as follows:

Oral Presentation, Abstract #53: Hydrodynamic injection through biliary system mediates therapeutic non-viral gene delivery in non-human primates
Session: Emerging non viral approaches for tissue and cell specific delivery
Session Date and Time: Tuesday, May 12, 2026, 10:15 AM ET

Poster, Abstract #2106: Hydrodynamic delivery of Cas9 ribonucleoprotein complex and plasmid DNA donor mediate therapeutic gene knock-in into the albumin locus of mice
Session Date: Wednesday, May 13, 2026

Poster, Abstract #2421: Non-viral gene therapy via hydrodynamic delivery through the biliary system yields therapeutic expression in porcine models of tyrosinemia and phenylketonuria
Session Date: Wednesday, May 13, 2026

Poster, Abstract #2519: Hydrodynamic injection through biliary system mediates human ATP7B DNA delivery in human-sized pigs to model treatment of Wilson Disease
Session Date: Wednesday, May 13, 2026

About HydroGene^TM

HydroGene is a biotechnology company focused on creating accessible gene therapies for all liver diseases. The company’s proprietary hydrodynamic delivery platform is localized, safe, direct injection into the liver, achieving durable and redosable expression. HydroGene has developed a broad pipeline of genetic medicines for rare diseases with plans to expand into common disorders. In pairing a routine clinical procedure with the simplest genetic medicine, HydroGene believes in a future where genetic administration could be a common and safe clinical procedure across hospitals and clinics. HydroGene has lab operations in Cambridge, MA. For more information, visit www.hydrogenetx.com

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info@hydrogenetx.com

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