ProQR Therapeutics

March 2021 Update: ProQR has published positive results from its Phase 1/2 Stellar trial of QR-421a, an investigational RNA therapy for the treatment of Usher syndrome and retinitis pigmentosa (RP) due to mutation(s) in exon 13 of the USH2A gene. Stellar study, Phase 1/2 clinical trial showed investigational RNA therapy QR-421a is effective and safe. ProQR plans to start final phase trials for people with USH2A mediated retinitis pigmentosa. With only one injection of QR-421a, the Stellar study showed benefit in the treated eye in comparison to the untreated eye across multiple eye tests. This benefit was seen across all participants in the Stellar study, which included clinical trial participants with both advanced vision loss as well as early to moderate vision loss. Based on these positive results, ProQR plans to conduct two final stage clinical trials or Phase 2/3 trials. ProQR will work with regulators to submit clinical trial protocols for two final stage registration trials called Sirius and Celeste. For more information on the safety & efficacy of the Stellar trial, please read ProQR’s Community Statement, QR-421a program update March 2021.

STELLAR or PQ-421a-001, is a first-in-human study that will initially include approximately 18 adults with vision loss due to mutations in exon 13 of the USH2A gene and will be conducted at about seven expert sites in North America and Europe. QR-421a is designed to exclude exon 13 from the USH2A mRNA, thereby removing the mutation in exon 13. This approach is also known as exon skipping. RNA is the "blueprint" for protein synthesis, and the skipping of exon 13 in the "blueprint" is expected to lead to a shortened but functional Usherin protein. 

ProQR Science News