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 or life threatening, and have limited treatment options. Amongst others we work on the development of new medicines for several forms of inherited blindness such as Leberâs congenital amaurosis, Usher syndrome type 2 and autosomal dominant retinitis pigmentosa.
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