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ProQR Announces Initial Pipeline Targets and Highlights Axiomer® RNA Editing Platform Technology at R&D Event
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"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 2025Axiomer activity demonstrated across multiple preclinical in vitro, organoid, and in vivo models; up to 50% editing observed in rodent and non-human primatesBeyond correction, broad applicability of Axiomer demonstrated in preclinical models with ability to modulate proteins by altering function, changing post-translational modifications, and modifying protein interactionsCompany reports YE 2022 financials; cash runway extended into mid-2026 following Eli Lilly milestone related to partnership expansionVirtual R&D event today at 10:00 am EDT/1600 CET LEIDEN, Netherlands & CAMBRIDGE, Mass., March 29, 2023 (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 initial pipeline programs focused on diseases that originate in the liver. ProQR will host a virtual R&D event today, during which the Company will showcase its proprietary Axiomer RNA-editing technology platform, detail the pipeline, and provide guidance on the advancement of programs toward the clinic. ProQR also reported its 2022 year-end financials and the extension of its cash runway guidance. \"Today’s R&D event highlights the important progress we have made to advance our proprietary Axiomer RNA editing platform technology and demonstrate its broad applicability, as we develop treatments for diseases with high unmet need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Along with our preclinical proof of concept data for the platform, a partnership with Eli Lilly that is exclusively focused on RNA editing, leading IP position, and cash runway into mid-2026, ProQR is leading the advancement of RNA editing as a new class of therapies for patients.\" Initial Pipeline Programs ProQR today announced AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs. These programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in indications with high unmet medi...