UgeneX Therapeutics

UgeneX Therapeutics is a Swiss biotechnology company dedicated to developing transformative gene therapies for patients with rare metabolic disorders, specifically focusing on Urea Cycle Disorders (UCDs). The company's lead program is a next-generation Adeno-Associated Virus (AAV) gene therapy for Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder. UgeneX's technology platform focuses on optimizing viral vectors to achieve high-level, durable enzyme expression while minimizing immunogenicity. As a spin-off from the University of Zurich and the University Children's Hospital Zurich, the company integrates clinical expertise in metabolic medicine with advanced genetic engineering. UgeneX aims to provide a definitive, one-time treatment for life-threatening conditions that currently require lifelong management and carry a high risk of neurological damage.