LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will p
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’
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
Data from ongoing Phase 1 study of AX-0810 in healthy volunteers demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohortsAX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the
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.