Drug intelligence / Profile preview

RAG-03

Development stage: Preclinical
Lead developer: Ractigen Therapeutics
Modality: Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics

Overview

RAG-03 is a small activating RNA (saRNA) drug candidate developed by Ractigen Therapeutics. Its mechanism of action involves stimulating the production of thrombopoietin (THPO) in the liver, which in turn enhances platelet count. The primary therapeutic goal is to counteract persistent thrombocytopenia, a condition characterized by abnormally low levels of platelets in the blood. As of May 2025, RAG-03 remains at the discovery stage and has not yet entered clinical trials or received regulatory approval[1][2][3].

Targets

TPO (Thyroid peroxidase)

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