Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
Stre
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas; initial pipeline targets to be disclosed in early 2023 with Analyst and Investor R&D event to be held in Q1 2023
Management Team appointments and p
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multi
… ProQR Announces Third Quarter 2023 Operating and Financial … NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through … said Daniel A. de Boer, Chief Executive Officer of ProQR. “We expect that the body of data on our platform and …
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
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 P
Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026Selected Development Candidates AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Ended 2025 with € 92.4 million cash and cash equivalents and achie
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 and pr
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones