ProQR Therapeutics is a biotechnology company listed on NASDAQ with offices in Leiden, the Netherlands and Cambridge, Massachusetts, USA. ProQR is focused on the development of life changing RNA therapies to meet the unmet need of individuals and families with common and rare diseases.
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025
Axiomer activity demonstrated across multiple preclinical in vitro
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
Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight dataMartin Maier, PhD nominated to Board with Annual General
In line with corporate strategy update in April, ProQR to focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas; update on initial pipeline targets expected in late 2022/early 2023
Additi
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3
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
Continued advancement of platform and initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1Further strengthened leading intellectual property (IP) position with issuance of new patent in the United S