Two Phase 2/3 clinical trials aim to validate ultevursen’s ability to stop vision loss in retinitis pigmentosa and Usher syndrome. Learn more about the trials here.
In a Phase 1/2 clinical trial investigational RNA therapy QR-1123 showed target engagement. ProQR plans to start a Phase 2 study for RHO mediated retinitis pigmentosa.
The clinical trial named Brighten evaluates the safety and tolerability of investigational RNA therapy sepofarsen in children with Leber congenital amaurosis.
A blog by Martin Hills from Retina UK living with Retinitis Pigmentosa on why research participation and genetic testing are essential in for drug development and how to get involved.
In a Phase 1/2 clinical trial investigational RNA therapy ultevursen (QR-421a) showed a benefit in vision. ProQR plans to start final phase trials for USH2A mediated retinitis pigmentosa.
The ProQR family is growing fast and we spoke to the Chief Medical Officer Aniz Girach who joined ProQR in 2019. We asked him about his impression of the company after he joined, as well as the new developments he’s been working on.
During a ProQR Expert Perspective call Dr. Girach (CMO at ProQR) discusses findings from an open label extension study of sepofarsen for LCA10 with Retinal Disease expert Dr. MacDonald (Prof Emer University of Alberta).
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
