… RNAtherapy Find out how RNAtherapy works, how it differs from … 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 …
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.
… diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Before founding ProQR, … In 1992 Dr. Valerio was appointed professor of gene therapy at the University of Leiden, where he also received …
… biotech company advancing our proprietary Axiomer RNA-editing platform technology. We are ProQR We are ProQR We … company dedicated to changing lives by developing RNA therapies for severe rare and common diseases. We focus … and Cambridge, Massachusetts, USA. Learn more about RNAtherapy and our Axiomer technology . We are on a mission We …
ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
