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
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