ProQR Therapeutics N.V.

ProQR Therapeutics N.V. is a clinical-stage biotechnology company based in Leiden, Netherlands, with a presence in Cambridge, Massachusetts, USA. The company, founded in 2012 by CEO Daniel A. de Boer, is dedicated to discovering and developing RNA therapeutics for severe genetic rare and common diseases, particularly those with no current treatment options. Its core business revolves around its proprietary Axiomer RNA editing platform technology, which aims to correct disease-causing mutations directly at the messenger RNA level.

The company's pipeline focuses on advancing RNA editing therapies. Its lead clinical program, AX-0810, targets NTCP for cholestatic diseases, with biliary atresia selected as the initial Phase 2 indication. Other programs in development include AX-0811 for cholestatic diseases, AX-0422 for Hurler syndrome, AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH), and AX-2402 for Rett syndrome. ProQR also maintains a strategic collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders.

Recent notable developments include the company's April 2026 investor event, where it highlighted pipeline expansion, including new programs AX-0811 and AX-0422, and announced a partnership with Ginkgo Bioworks for AI-enabled discovery. ProQR expects target engagement data for AX-0810 in the first half of 2026 and has a cash runway extending into mid-2027. In February 2026, the company announced board changes, with co-founder Dinko Valerio and Alison Lawton stepping down. Daniel A. de Boer continues to lead as Founder and CEO, a role he has held since the company's inception. Analysts generally hold a "Moderate Buy" consensus rating for ProQR Therapeutics.