ProQR Therapeutics 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." ProQR 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 medical need, the ability to leverage the existing proven delivery technology to the liver, the opportunity to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept, and the availability of well-defined clinical endpoints.
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