Patients & Community Acting in the interest of patients

Living with Inherited retinal disease - Maartje de Kok and her family

ProQR is dedicated to developing RNA therapies to improve the lives of patients, families and communities living with inherited retinal diseases, a group of rare diseases causing blindness. To achieve this goal, 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.

Our patient-focused approach

  • We are committed to the communities we serve.
  • We value patients as experts and strive to integrate their insights into our decision-making.
  • We act with a sense of urgency and purpose in the interest of patients.
  • We believe in early, consistent and transparent engagement with the patient community. 

If you have questions or want to stay informed please contact patientinfo@proqr.com or sign up for our Eye Connect newsletter.

ProQR clinical trials for genetic eye diseases

Here is a list of all our ongoing clinical trials.
If you are not sure what a clinical trial is all about? Read this first.

Brighten (ongoing, recruiting)

Clinical trial of sepofarsen for children with CEP290 mediated Leber congenital amaurosis

Read about the Brighten trial

Brighten logomark

Fuchs Focus (ongoing, recruiting)

Phase 1b clinical trial of QR-504a, an investigational RNA therapy for TCF4 mediated Fuchs endothelial corneal dystrophy. This trial is recruiting individuals that have FECD due to trinuclear repeat expansion mutations in the TCF4 gene.

Read about the Fuchs Focus trial

Fuchs Focus logomark

Sirius (planned, not yet recruiting)

Phase 2/3 clinical trial for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will include adults (age 18 and up) that have advanced vision loss.

Read about QR-421a

Sirius logomark

Celeste (planned, not yet recruiting)

Phase 2/3 clinical trial for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will include adults (age 18 and up) that have early to moderate vision loss.

Read about QR-421a

Celeste logomark

How does RNA therapy work?

Learn how the latest RNA technologies can help treat rare genetic diseases