ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-1123.
ProQR plans to start enrolling patients in a Phase 1/2 trial for QR-1123 in 2019.
QR-1123 is a first-in-class investigational oligonucleotide designed to address the underlying cause of the vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.
P23H is the most prevalent mutation associated with adRP in the U.S. This disease causes progressive vision loss in approximately 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies for adRP and QR-1123 is the first investigational medicine to be developed for patients that suffer from this disease.
‘We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,’ said Daniel A. de Boer, Chief Executive Officer of ProQR. ‘This represents our fifth IND in less than five years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.’
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