HydroGene

Worldwide, over 4M patients above the age of 25 are affected by liver-based monogenic hereditary disorders. Hemophilia A, affecting roughly 20K males in the United States, is a burdensome liver-based genetic condition. The current standard of care for these patients is a thrice-weekly infusion of blood clot supplements to manage chronic bleeds, resulting in annual medical costs over $300K. Hospitalizations for acute bleeds have resulted in an additional $1M annually upon the patient. Gene therapy has been proposed as a potential cure for these costly and difficult-to-manage conditions, as it can replace a missing or defective gene and treat the disease. Traditionally, gene therapy is delivered through the bloodstream via adeno-associated viral vectors (AAVs). However, AAVs can trigger an immune response, as many patients have preexisting antibodies that fight AAVs. Additionally, due to its small genome, AAVs require complex and expensive bulk manufacturing systems. An alternative to viral delivery, HydroGene’s novel non-viral system integrates into a common endoscopy procedure that accesses liver cells via the biliary ducts, a delivery site well supported in literature. While traditional gene therapy lacks the ability to target specific tissues, HydroGene provides a localized delivery that maximizes safety and efficacy and minimizes procedural costs. Additionally, HydroGene’s technology is versatile and applicable to an array of monogenic disorders and compatible with a wide range of therapeutics, including DNA, RNA, and protein-based medicines. With a simpler, safer method of gene delivery, HydroGene aims to accelerate the progress and expand the reach of gene therapy research.

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