… to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare … advisory board. "Dr. Yu is a pioneer of targeted RNAediting to correct protein translation defects caused by …
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 …