Our broad pipeline of investigational RNA therapies is evolving, and we are currently conducting several clinical trials. Find below the clinical trials for which we are recruiting patients.
COVID-19 vaccine statement
There is no contraindication for patients who have taken part or wish to take part in the ProQR sponsored studies to receive the COVID-19 vaccine in combination with antisense oligonucleotides including our investigational RNA therapies. The protocol doesn’t exclude patients who are enrolled in studies from receiving the vaccine or who may have received it in the past or who may receive it in the course of any ProQR sponsored study.
Phase 2/3 clinical trial of sepofarsen, an investigational RNA therapy for CEP290 mediated Leber congenital amaurosis. This trial is recruiting adults and children (age 8 and up) that have LCA10 due to the p.Cys998X (c.2991+1655A>G) mutation in the CEP290 gene.
Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial is recruiting adults (age 18 and up) that have RP due to mutations in exon 13 of the USH2A gene.
Phase 1/2 clinical trial of QR-1123, an investigational RNA therapy for RHO mediated retinitis pigmentosa. This trial is recruiting adults (age 18 and up) that have RP due to the P23H (c.68C>A) mutation in the RHO gene.