ProQR Sets Q2 2026 AX-0810 Data Readout, Expands Pipeline with AX-0811 and AX-0422
ProQR expects target engagement data for AX-0810 in healthy volunteers by Q2 2026 and has selected biliary atresia as its initial Phase 2 indication. The company plans CTA filings for AX-0811 in mid-2026 and AX-0422 in early 2027, bolstering its Axiomer RNA editing pipeline.
1. AX-0810 Target Engagement
AX-0810, ProQR’s lead Axiomer RNA editing candidate targeting NTCP, remains on track to report target engagement data in healthy volunteers by Q2 2026. The company has designated biliary atresia for its initial Phase 2 study based on strong biological rationale and high unmet need.
2. Pipeline Expansion
ProQR plans a CTA submission for AX-0811, a next-generation NTCP candidate, in mid-2026 with first clinical data due by year-end, and expects an early-2027 CTA filing for AX-0422 targeting IDUA in Hurler syndrome. The company also outlined plans for an investigator-initiated PNPLA3 program trial in China in H1 2027.
3. Axiomer Platform Enhancement
ProQR described its AI-enabled discovery engine and high-throughput screening partnership with Ginkgo Bioworks to accelerate RNA editing therapeutic design and optimization across multiple programs.
4. Financial Runway and Catalysts
ProQR’s current cash runway extends into mid-2027, supporting multiple clinical data readouts across its pipeline—including AX-2911 and other candidates—without near-term financing needs.