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AX-003

Development stage: Preclinical
Lead developer: Axelyf
Modality: mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration: Intravenous

Overview

AX-003 is a preclinical mRNA-based therapeutic being developed by Axelyf for the treatment of autoimmune hepatitis (AIH). The drug utilizes a lipid nanoparticle (LNP) delivery system designed for selective liver targeting. Once delivered to liver cells, the mRNA encodes a specific immune checkpoint protein intended to modulate and dampen the localized autoimmune response. By leveraging the liver's natural role in immune tolerance and using selective delivery, AX-003 aims to restore immune homeostasis in patients with AIH. As of 2024, the program is in the research stage, with clinical trials anticipated to begin in early 2028.

Other names: AX-003AX003AX 003

Targets

Checkpoint (Immune checkpoints)

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