Clinical trials

Clinical trials

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.

Illuminate - sepofarsen for CEP290 LCA

Illuminate

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.

Read about the Illuminate trial

Illuminate logomark
Stellar - QR-421a for USH2A RP

Stellar

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.

Read about the Stellar trial

Stellar logomark
Aurora - QR-1123 for RHO RP

Aurora

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.

Read about the Aurora trial

Aurora logomark