Top-line data from pivotal Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated LCA10 anticipated late Q1/early Q2 2022
Five-target collaboration with Lilly highlights significant potential of ProQR’s Axiomer® RNA base-editing pla
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators and pr
Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome
… to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today … to progress its other pipeline candidates, to advance its RNA-editing technology platforms, and for working capital and …
… bring a tremendous amount of experience in the fields of RNA-based therapeutics and genetic pediatric diseases. The … of the Company’s novel and proprietary next generation RNAediting technology, Axiomer®, that utilizes endogenous …
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022
Positive data reported from the Phase 1/2 clinical trial of QR-421a for the treatment of Usher syndrome and non-syndromic retini
… company dedicated to changing lives through transformative RNA therapies, today announced that the Company will host a … will highlight its proprietary Axiomer™ ADAR-mediated RNAediting platform, along with updates on its pipeline of …