Nephrogen is at the forefront of developing curative gene therapies specifically targeting life-threatening kidney diseases, with a primary focus on autosomal dominant polycystic kidney disease (ADPKD). Their innovative approach integrates CRISPR-Cas genome editing with advanced nucleic acid delivery systems, such as adeno-associated viruses (AAVs) and lipid nanoparticles (LNPs). This combination allows for a targeted intervention at the genetic level, aiming to halt the progression of ADPKD and potentially provide a complete cure. Nephrogen's technology is characterized by a non-invasive delivery method that ensures maximum therapeutic effect, alongside engineered AAVs that exhibit significantly improved efficiency in targeting kidney tissues compared to existing options.

In addition to their gene therapy initiatives, Nephrogen employs a rapid drug discovery platform that utilizes cutting-edge innovations in computer vision and high-throughput screening (HTS). This platform is designed to identify new small molecule drugs for various cystic kidney diseases, including nephronophthisis. By pursuing unique gene targets within the PKD1 gene, Nephrogen is committed to a personalized approach in their therapeutic development, which is supported by a robust patent portfolio that facilitates foundational partnerships and transformative treatment options for patients suffering from genetic kidney diseases.

United States

Nephrogen is a therapeutics company developing transformative medicines for kidney disease. Their initial focus is on gene therapy for autosomal dominant polycystic kidney disease (ADPKD).

Nephrogen

Company Details