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.
At ProQR science is literally at the heart of what we do. Our passion lies in discovering the latest RNA technologies and translating them to life-changing treatments.
At ProQR, we are dedicated to improving the lives of patients and families affected by rare and common disorders using Axiomer, our innovative RNA editing platform technology.
At our offices in Leiden we are advancing our proprietary Axiomer RNA-editing platform technology. Our ProQRians, representing over 30 nationalities, are passionate and driven to change the lives of patients and their loved ones.
Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseasesInitiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagemen
… transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced … section of the ProQR website at www.proqr.com . About Axiomer™ ProQR is pioneering a next-generation RNA base …
… momentum toward the clinic Accelerated development of Axiomer™ for CNS applications via new research partnership … on RNA editing, ProQR made important progress with Axiomer, our ADAR-mediated RNA editing technology platform,” …
