We are ProQR

We are ProQR, a company on a mission to create new medicines for patients in need. We focus on a group of diseases called rare diseases, with a very high need for new medicines. Out of the approximately 7,000 rare diseases, less than 400 have a treatment, and we intend to change that. To treat these diseases we use RNA therapies, a technology that allows us to address inherited diseases that are caused by a genetic defect.

Our focus is on diseases that are very severe and have limited treatment options. Amongst others we work on the development of new medicines for several forms of inherited blindness such as Leber’s congenital amaurosis, Usher syndrome and autosomal dominant retinitis pigmentosa.


Play “How do ProQRs RNA therapies work” on YouTube (English subtitles available)

Patients come first

We know first-hand what impact rare disease make to patients and their loved ones, and therefore we always put patients first. Our mission is to help patients by creating new medicines to address their disease. As we believe in passion and commitment we have built a team of 140 ProQRians, from all walks of life and over 30 different nationalities, which are up to the challenge committed to make a difference to the patients we serve.

Our science

Cutting edge science is at the core of the medicines we develop. At our state-of-the-art labs in Leiden, the Netherlands, our scientists are working day in day out to discover and optimize ways to treat genetic rare diseases. We are specialized in the development of RNA therapeutics; drugs that consist of a short piece of synthetic RNA. This technology enables us to make temporary corrections to “genes” and therefor restore protein expression that is crucial for our bodies to function. Since the start of our company we have invented and optimized an arsenal of different RNA technologies that each are able to address a different type of genetic defect.

Together we are stronger

To accomplish our mission we have created a global network of partners: patients, patient organizations, scientist, doctors and regulators. In collaboration with these partners we are able to better understand the need of patients, advance our science and accelerate our development programs to get new medicines to patients in need as rapid as possible. As we believe in the power of collaboration, we work closely with communities and experts to accomplish our mission for the patients we serve.

Founded in 2012

When our CEO was confronted with the diagnoses of his newborn son with cystic fibrosis, he decided to start ProQR to help patients like his son. He collected a group of experienced biotech entrepreneurs around him and in 2012 founded ProQR with co-founders Dinko Valerio, Gerard Platenburg and Henri A. Termeer.

Henri was a visionary rare disease leader that pioneered rare disease drug development as the CEO of Genzyme. In 2017 Henri unexpectedly passed away. ProQR will continue to honor his legacy through completing the mission we set out together.
Learn more on HenriTermeer.org

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