… In November, the first patient was dosed in the Phase 1/2 open-label trial (PQ-110-001) to assess the safety, … six adults (≥ 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. During … expertise in genetic retinal disease in the U.S. and Europe. The objectives of the trial include safety, …
Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 anticipated in 2024, building further momentum toward the clinicAccelerated development of Axiomer™ for CNS ap
Divestment of sepofarsen and ultevursen ophthalmic programs supports ProQR’s strategic focus on Axiomer® RNA editing platform technology and continued advancement of pipeline
€129 M cash and cash equivalents as of June 30, 2023 providing runway into mid-2026
LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 18, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today hosted a virtual Analyst Event.
… biopharmaceutical company engaged in the discovery and development of RNA-based therapeutics for the treatment of … its Therapeutic Development Network, a network of CF key opinion leaders, as well as to an extensive clinical trial …