The FDA has cleared an investigational new drug application for QR-421a for Usher syndrome type 2, ProQR Therapeutics announced in a press release.
QR-421a, an investigational RNA-based oligonucleotide, addresses the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene, the release said.
The company plans to begin the phase 1/2 STELLAR clinical trial of QR-421a in early 2019. The first-in-human, double-masked, international, randomized study will include approximately 18 adults, with preliminary data expected in mid-2019.
“We are pleased to be advancing QR-421a, our secondary therapy for an inherited retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines for severe genetic rare diseases,” ProQR CEO Daniel A. de Boer said in the release.