At ProQR science is literally at the heart of what we do. Our passion lies in discovering the latest RNA technologies and translating them to life-changing treatments.
At our offices in Leiden we are advancing our proprietary Axiomer RNA-editing platform technology. Our ProQRians, representing over 30 nationalities, are passionate and driven to change the lives of patients and their loved ones.
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
Stre
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3