We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.
We are ProQR
We are on a mission
From the day our company was founded in 2012, our mission has been to help people living with rare diseases that currently have no treatment options. This remains the sole focus of our company today.
Read more about our Founding story.
RNA therapies to stop or reverse vision loss
We believe that the best way to fulfill this mission is to develop RNA therapies for genetic eye diseases. We have built a platform to develop these highly targeted therapies to address the underlying cause of the disease and improve vision. For the vast majority of the more than 2 million people living with an inherited retinal disease worldwide, there is no treatment available for the vision loss and eventual blindness that can occur. And with more than 300 genes identified that cause inherited retinal diseases, there is an immense opportunity to develop therapies for people in need.
What is RNA therapy?
Learn about our mission to help treat rare genetic diseases
Patient-focused drug development
In order to achieve our goals, ProQR strives to integrate the patient voice into our decision-making throughout the drug development process as we believe that a patient-focused strategy is crucial to our success.
Read more about what we do for patients and the broader inherited disease community on Patients & Community.