ProQR’s Usher syndrome candidate shows positive interim data

Interim results of a phase 1/2 clinical trial of QR-421a has shown early evidence of activity in Usher syndrome, with 25% of patients experiencing a benefit across outcome measures, ProQR Therapeutics announced in a press release.
The first-in-human, double-masked, randomized, 24-month Stellar study is evaluating the safety and efficacy of a single intravitreal injection of several dose levels of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene.
Six subjects were enrolled in the low-dose 50 µg cohort, with four receiving treatment (Read more...)

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