ProQR Therapeutics is a clinical-stage biotech developing RNA editing therapies using its proprietary Axiomer platform. Rather than permanently altering DNA like CRISPR or gene therapy, Axiomer uses synthetic molecules called editing oligonucleotides (EONs) to make targeted, reversible edits at the RNA level by recruiting naturally occurring ADAR enzymes. ProQR argues this reversibility offers safety and dosing flexibility advantages over permanent gene-editing approaches. The pipeline focuses on liver diseases and CNS/neurological disorders. The lead program, AX-0810, targets cholestatic liver diseases (PSC and biliary atresia) and is currently in Phase 1 trials, with biomarker data expected in the first half of 2026. AX-2402 targets Rett syndrome (partially funded by a $9.2M grant from the Rett Syndrome Research Trust) and is headed for a first-in-human trial in 2027. AX-2911 targets a genetic driver of MASH, and AX-1412 is an early-stage cardiovascular program. ProQR has no approved products and funds operations through equity raises, grants, and its collaboration with Eli Lilly, signed in 2021, under which ProQR received $125M upfront and is eligible for up to ~$3.75B in milestones plus royalties. ProQR's strategy is to advance wholly-owned programs to clinical proof-of-concept while licensing the Axiomer platform to large pharma partners for targets it cannot pursue independently.