Drug intelligence / Profile preview

RAG-05

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

Overview

RAG-05 is a small activating RNA (saRNA) drug candidate developed by Ractigen Therapeutics. Its mechanism of action is to stimulate the activity of porphobilinogen deaminase (PBGD), an enzyme involved in heme biosynthesis. By upregulating PBGD, RAG-05 aims to address metabolic deficiencies associated with acute intermittent porphyria, a rare genetic disorder characterized by defects in heme production. The drug is currently at the discovery stage and has not yet entered clinical trials[1].

Targets

Hydroxymethylbilane synthase promoter (HMBS promoter)

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