ProQR Therapeutics (PRQR) The Citizens Life Sciences Conference 2026 summary

Strategic focus and technology platform

• Since 2022, the company has focused exclusively on developing its proprietary Axiomer RNA editing technology, pioneering ADAR-mediated RNA editing with a broad, homegrown IP estate.

• The approach targets diseases where RNA editing offers advantages over other modalities, prioritizing indications with high unmet need and limited or no current treatments.

• The technology enables transient, reversible protein modification without permanent genetic changes, aiming for safer, more controllable therapies.

• Delivery strategies leverage established methods: GalNAc conjugation for liver targets and intrathecal administration for CNS indications.

Lead program and clinical development

• Lead program AX-0810 targets NTCP in the liver for cholestatic diseases, aiming to block bile acid uptake while preserving other transporter functions.

• Early phase I data in healthy volunteers showed expected pharmacokinetics and no safety signals; three biomarkers are being tracked for target engagement.

• Three dose levels (3, 6, 9 mg/kg) are being tested, with data from all healthy cohorts expected in the first half of the year.

• Success in healthy cohorts will lead to a patient cohort under the same protocol, with subsequent indication selection between PSC and BA.

• The study is single-center, based in the Netherlands, designed for streamlined progression.

Pipeline and future plans

• Rett syndrome program has selected a clinical candidate, with IND-enabling studies underway and first-in-human trials planned for the first half of 2027.

• A third program targets PNPLA3 for fatty liver disease (MASH), with further development plans to be announced later this year.

• The platform's chemistry and oligo modifications are repeatable, and clinical success in one program is expected to have positive read-through for others.

• The company maintains a wholly owned pipeline while leveraging partnerships, notably a large collaboration with Lilly covering 10 targets and providing significant non-dilutive funding.