#United States #Boston #gene therapy #ASGCT #HydroGene

Groundbreaking Advances in Non-Viral Gene Therapy

HydroGene Therapeutics is set to showcase its latest advancements in non-viral DNA delivery methods during the upcoming 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting from May 11-15 in Boston, Massachusetts. The biotechnology company focuses on developing accessible gene therapies for various liver diseases. The significance of their research lies in its potential to revolutionize gene therapy approaches that have historically relied heavily on viral methods.

The cornerstone of HydroGene's innovation is its unique non-viral delivery system for gene therapies that are both safe and durable. This technology enables gene expression levels comparable to existing adeno-associated virus (AAV) treatments, while also addressing the multiple limitations associated with viral methods including toxicity and high manufacturing costs. HydroGene's core method involves hydrodynamic injection, which takes advantage of hydraulic pressure to deliver naked DNA directly to the liver cells in a highly efficient and organ-specific manner. This delivery approach allows for a robust therapeutic effect without the challenges posed by viral vectors.

During the ASGCT meeting, HydroGene will present initial findings from non-human primate (NHP) models, demonstrating not only the safety of their gene delivery method but also the longevity and reproducibility of the therapeutic expression. Notably, the results indicate that DNA delivery into the liver can yield stable expression of therapeutic genes for over six months, a promising indicator of the potential for practical human applications. Co-founder Robert Kruse, MD, PhD, remarked on the achievement, saying, “Our earlier work at Johns Hopkins University established the viability of safely delivering DNA into large animal models. We are incredibly excited to extend this progress to non-human primates, which serve as the gold standard for gene therapy studies.”

In addition to the oral presentation, HydroGene will display multiple poster presentations showcasing its advancements in treating rare genetic disorders such as Wilson disease, phenylketonuria, and hereditary tyrosinemia. These posters underline the efficacy of their hydrodynamic delivery in genetic models, illustrating not only the ability to achieve high levels of gene transfection but also the repair of relevant metabolic biomarkers. These milestones are crucial for future clinical trials and potential therapeutic interventions in human patients suffering from these conditions.

HydroGene's research reflects a shift toward safer and more accessible gene therapy options. As Vivek Kumbhari, MBChB, PhD and another co-founder of HydroGene, noted, this technology could streamline the process for genetic medicine by integrating it with routine outpatient procedures, thus reducing the burden of traditional interventions that often involve extended periods of immunosuppression and other significant side effects.

The company’s innovative approach employs the biliary system for administering their DNA delivery, leveraging established medical techniques such as endoscopic retrograde cholangiopancreatography (ERCP) to navigate direct access to the liver. This novel delivery mechanism not only optimizes the effectiveness of their therapies but also underscores HydroGene's commitment to developing non-invasive techniques that can be utilized across various hospitals and clinics worldwide.

Any further discussions at the ASGCT will likely center on HydroGene's plans for upcoming first-in-human trials in 2027. These trials aim to validate their non-viral delivery approach in patients diagnosed with hemophilia, providing an opportunity to address a significant clinical need with a novel solution. HydroGene’s approach represents hope for many seeking more effective and less invasive treatments for genetic liver diseases.

With a focus on developing a broad pipeline of genetic therapies for rare disorders and aspirations of addressing common diseases in the future, HydroGene is setting the stage for a new era in genetic medicine. Founded in 2022 and emerging from stealth, the company has already garnered support from notable partners like Curie.Bio and Liquid2 Ventures, along with collaborations with leading institutions such as Johns Hopkins and Mayo Clinic.

For more information about HydroGene's breakthroughs in gene therapy, check their website at HydroGene.