… RNA therapy Find out how RNA therapy works, how it differs … underlying cause of the disease. Watch: How do ProQR's RNA therapies work? A short introduction to RNA therapies Why focus on RNA therapies? Why focus on RNA therapies? RNA therapies can …
… Board with Leaders in Inherited Retinal Disease and RNA Therapy LEIDEN, Netherlands & CAMBRIDGE, Mass., July 21, … to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today … of leading experts in inherited retinal disease and RNA therapies,” said Daniel A. de Boer, Chief Executive …
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
… a rare genetic disorder, with the objective to develop RNA therapies for rare diseases. Prior to founding ProQR, … Society and the Scientific Advisory Board of the Gene and RNA Therapy Center Tübingen, Germany. During his 25 years of … Society and the Scientific Advisory Board of the Gene and RNA Therapy Center Tübingen, Germany. During his 25 years of …
… company dedicated to changing lives by developing RNA therapies for severe rare and common diseases. We focus … and Cambridge, Massachusetts, USA. Learn more about RNA therapy and our Axiomer technology . We are on a mission …
Positive preliminary data from InSight extension study of sepofarsen for LCA10 reported – consistent with benefit seen in Phase 1/2 and building confidence in Phase 2/3 Illuminate trial;
Strategic convertible debt financing extends runway into 2023 if fully draw
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in
Illuminate Phase 2/3 trial of sepofarsen expected to complete enrollment in Q1 2021; additional data from Phase 1/2 InSight extension study to be reported in H2 2021
Enrollment completed for QR-421a dosing cohorts and data from next Phase 1/2 interim
… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today hosted a … Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber … to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare …
Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;Updated data from the Phase 1/2 InSight extension st
