The year 2016 was important for ProQR. We completed a clinical trial in patients with cystic fibrosis and received Fast Track Designation for the program. We advanced our programs for Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa further through pre-clinical development and significantly expanded the team.
ProQR was born from the dream to beat cystic fibrosis in just one child. Now ProQR has one goal. Use groundbreaking science to develop medicines that will transform the lives of patients with severe genetic disorders and their loved ones.
Play the movie to learn more about
what drives us at ProQR.
Applying top-notch science to develop drugs for patients in need is what drives us. With an ingenious and persistent team of pioneers we love to welcome people who can accelerate our mission. Do you think you are ready to become a ProQRian? Let us know! Read more
Severe Genetic Disorders
More than 70,000 patients worldwide suffer from cystic fibrosis, a genetic disease with a limited life expectancy. Leber’s congenital amaurosis affects approximately 15,000 patients in the Western world and there is currently no disease modifying treatment available for this blinding disease. Learn more about our missions and how we aim to change the course of severe genetic disorders. Read more