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First patient dosed in ProQR trial of QR-421a for Usher syndrome

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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 with early but promising data for sepofarsen in patients with [Leber’s congenital amaurosis 10],” Daniel A. de Boer, CEO of ProQR, said in the release.

Interim data from the trial are expected mid-year.