ProQR has dosed the first patient in a phase 1/2 trial of QR-421a for Usher syndrome type 2 or non-syndromic retinitis pigmentosa, according to a press release.
The double-masked, randomized, multisite STELLAR trial will include 18 adult patients who have vision loss due to mutations in exon 13 of the USH2A gene.
Patients will receive an intravitreal injection of QR-421a, an RNA-based oligonucleotide, or sham.
“Usher syndrome is a devastating disease, so we are pleased to advance QR-421a into the clinic with the goal to make a difference for these patients, similar to what we have observed
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