ProQR Therapeutics

PRQR Rare disease Lead: Phase 1 2 Biotech Distilled articles ↓

Most recent annual filing: 2026-03-12

Catalyst timeline

AX-0810 Ph1 readoutby year-end 2026AX-0810 Ph1 readout, by year-end 2026, major
General corporate updateSep 30, 2026General corporate update, Sep 30, 2026, routine
AX-0811 Ph1 interimJan 2027AX-0811 Ph1 interim, Jan 2027, major
AX-0810/biliary atresia IIT readout1H 2027AX-0810/biliary atresia IIT readout, 1H 2027, material
AX-0422 readout1H 2027AX-0422 readout, 1H 2027, material
AX-0422 filingearly 2027AX-0422 filing, early 2027, material
AX-2911 trial start1H 2027AX-2911 trial start, 1H 2027, routine
AX-0810/biliary atresia Ph2 startMid-2027AX-0810/biliary atresia Ph2 start, Mid-2027, notable

Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.

Pipeline (7 active programs)

AX-2402

Nucleic Acid Target: MECP2

Rett Syndrome Preclinical Rett Syndrome Research Trust

AX-0810

Nucleic Acid Target: NTCP

Cholestatic Diseases Phase 1 subcutaneous
Biliary Atresia

AX-2911

Nucleic Acid Target: PNPLA3

Metabolic Dysfunction-Associated Steatohepatitis Preclinical

AX-1412

Nucleic Acid Target: B4GALT1

Undisclosed indication Preclinical

AX-0811

Nucleic Acid Target: NTCP, SLC10A1

Cholestatic Diseases Phase 1

AX-0422

Nucleic Acid Target: IDUA

Hurler Syndrome

Technology platforms

Axiomer

Next-generation RNA base editing technology using ADAR (Adenosine Deaminase Acting on RNA) machinery; Editing Oligonucleotides (EONs) mediate single nucleotide A-to-I changes in RNA to correct disease-causing mutations, modulate protein expression, or alter protein function; applicable to both rare and prevalent diseases with unmet need.

Targets: ADAR, NTCP, IDUA, PNPLA3

Axiomer · Eli Lilly

Next-generation RNA base editing technology using endogenous ADAR machinery to make single nucleotide A-to-I edits in RNA; applicable to both rare and prevalent diseases with unmet need

Targets: ADAR

Axiomer · Eli Lilly and Company

Next-generation RNA base editing technology using endogenous ADAR machinery to make A-to-I single nucleotide edits in RNA; designed to correct disease-causing mutations, modulate protein expression, or alter protein function; applicable to both rare and prevalent diseases including cholestatic liver diseases, MPS I Hurler syndrome, and MASH.

Targets: ADAR, NTCP, IDUA, PNPLA3, B4GALT1

Axiomer · Ginkgo Bioworks

Next-generation RNA base editing technology using ADAR (Adenosine Deaminase Acting on RNA) to make single nucleotide edits in RNA via Editing Oligonucleotides (EONs); applications across multiple tissues and disease areas including rare and prevalent diseases. Integration of AI-enabled discovery and high-throughput screening to accelerate design and optimization.

Targets: ADAR

Biotech Distilled coverage (2)

2026-09-10 UPDATE: ProQR Therapeutics (PRQR) - The Gate Opened, and the Clock Moved 🔒 Paid
2026-09-01 ProQR Therapeutics (PRQR) - Scientific Deep Dive for AX-0810, AX-0811 and the Axiomer RNA Editing Pipeline 🔒 Paid

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.