Targeting rare diseases with RNA treatments


There are approximately 7,000 known rare diseases, however, less than 6% of these have a treatment. It is estimated that between 25–35 million Americans live with a rare disease. The company ProQR is dedicated to developing new therapeutics for patients living with untreated rare diseases.

We caught up with Daniel de Boer, Chief Executive Officer at ProQR to learn more about the company’s approach to finding new treatments for rare genetic diseases.

Q: Could you tell us more about ProQR, the company history, mission and goals?

A: At ProQR we develop RNA medicines for rare genetic diseases. I started ProQR in 2012 because my son was diagnosed with a rare genetic disease for which there was no treatment. We now have a diversified pipeline of programs for severe genetic eye and skin disorders with the goal to have a positive impact on the lives of patients and their loved ones.

Related article:  Using genetics and social media to find answers for rare 'undiagnosed' diseases

ProQR recently announced results from a planned interim analysis of its Phase 1/2 trial of QR-110 in patients with Leber congenital amaurosis 10.

Q: What other drug candidates are currently in development and what indications are they being investigated for?

A: We have a broad pipeline of RNA medicines targeting severe genetic rare diseases. Besides QR-110 for LCA10 we are developing several other programs for genetic eye disorders including Usher syndrome, Stargardt’s disease and Fuchs endothelial corneal dystrophy.

Read full, original post: Developing RNA Medicines for Rare Genetic Diseases

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