Category: Approval

January 3, 2019 Off

ProQR’s QR-421a fast tracked, Phase 1/2 clinical trial in 2109

By Dino Mustafić

ProQR Therapeutics N.V. got Fast Track designation from the Food and Drug Administration (FDA) for QR-421a, a first-in-class investigational RNA-based oligonucleotide to tackle the underlying cause of the vision loss related to the Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.