… 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