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AX-2911
- Development stage
- Preclinical
- Lead developer
- ProQR Therapeutics
- Modality
- Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
AX-2911 is an investigational oligonucleotide drug developed by ProQR Therapeutics using its proprietary Axiomer RNA editing platform. It is designed as a hepatoprotectant and functions as an RNA modulator targeting the PNPLA3 gene (adiponutrin) for the treatment of metabolic diseases, specifically non-alcoholic steatohepatitis (NASH), also known as metabolic dysfunction-associated steatohepatitis (MASH). The mechanism involves modulation of adiponutrin expression through ADAR-mediated RNA editing, aiming to correct disease-driving genetic variants at the RNA level. As of March 2025, AX-2911 remains in preclinical development with plans for clinical candidate selection and anticipated entry into Phase I trials in 2026[1][2][3][5][10].
02
Targets
PNPLA3 (Patatin-like phospholipase domain-containing protein 3)
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