ProQR: Developing RNA Therapies for Rare Genetic Disorders

There is a great need for transformative treatments that increase life expectancy and improve the quality of life of patients and families living with rare inherited diseases. Current therapeutic approaches, including small molecules and enzyme replacement therapies, have limitations and cannot be applied to every disease. ProQR specializes in the development of RNA therapies and is expanding its toolbox of RNA approaches to develop life-changing medicines for rare genetic disorders that are currently untreatable.