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Usher syndrome candidate receives orphan drug designation

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The FDA and the European Medicines Agency have granted orphan drug designation to QRX-421, an investigational drug for Usher syndrome, according to a press release from ProQR Therapeutics.

The investigational RNA-based oligonucleotide addresses the mutations in exon 13 of the USH2A gene, which can cause loss of functional USH2A protein, causing Usher syndrome, the release said. The drug excludes exon 13 from the mRNA and produces functional USH2A protein.

“We are pleased to have ODD designation for both our programs targeting Usher syndrome in the U.S. and EU, representing yet another important milestone for our company and highlighting the unmet need for patients in this disease,” David M. Rodman, MD, ProQR chief development strategy officer, said in the release. “At ProQR, we are focused on designing accelerated development strategies that capitalize on our oligonucleotide approach to potentially bring our novel medicines to patients quicker and receiving ODD designations for these is an important step towards this goal.”