ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing OligonucleotidesPreclinical proof of concept for the Company’
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.