ProQR Unveils Preclinical AX-0422, AX-2402, AX-2911 Data and AX-0810 Clinical Preview
ProQR will present data on AX-0422 in Hurler syndrome, AX-2402 in Rett syndrome and AX-2911 for hepatic steatosis at TIDES USA, ASGCT, RNA Society and EASL conferences in May. Its lead program AX-0810 is advancing toward initial clinical data in healthy volunteers later this quarter.
1. Upcoming Scientific Presentations
ProQR will present five oral and poster sessions at four major conferences in May, including TIDES USA (May 11–14), the American Society of Gene and Cell Therapy Annual Meeting (May 11–15), the RNA Society Annual Meeting (May 26–31) and the EASL Congress (May 27–30). Gerard Platenburg and other team members will deliver updates on multiple pipeline programs and platform developments.
2. Preclinical Data Highlights
Preclinical data to be showcased include AX-0422 targeting IDUA in Hurler syndrome, which demonstrated multi-organ glycosaminoglycan reduction and improved functional outcomes versus enzyme replacement; AX-2402 in Rett syndrome, showing phenotype reversal and RNAseq-confirmed pathway modulation; and AX-2911 converting PNPLA3 I148M variant to wild-type-like form, markedly reducing hepatic steatosis in vivo.
3. AX-0810 Clinical Readout
ProQR's lead candidate, AX-0810, which targets NTCP for cholestatic diseases, is advancing through a Phase 1 study in healthy volunteers, with initial clinical data expected later this quarter. This upcoming readout will inform safety and therapeutic potential for cholestatic conditions and support further development decisions.
4. Axiomer Platform Advancements
In addition to program-specific data, ProQR will highlight advances in its Axiomer RNA editing platform, including optimized oligonucleotide designs for improved cellular uptake, endosomal escape and ADAR recruitment to enable precise base editing across liver and CNS targets.