First patients dosed in phase 2/3 gene therapy trial for retinitis pigmentosa

The first patients have been dosed in the phase 2/3 Sirius and Celeste clinical trials investigating QR-421a in patients with USH2A mediated retinitis pigmentosa and Usher syndrome, according to a press release from ProQR Therapeutics.

The double-masked, randomized, sham-controlled, 24-month, multiple-dose studies are investigating the safety and effectiveness of QR-421a. Both studies will enroll patients 12 years and older, who have vision loss attributed to mutations in exon 13 of the USH2A gene, in one of three arms. Patients in the first arm will receive a 180 µg loading dose followed by 60 µg maintenance doses, patients in the second arm will receive a 60 µg loading dose followed by 60 µg maintenance doses, and patients in the third arm will serve as a control.


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