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RAG-12

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

Overview

RAG-12 is a small activating RNA (saRNA) drug candidate developed by Ractigen Therapeutics. It is designed to address haploinsufficiency of the SERPING1 gene, which leads to C1 esterase inhibitor (C1-INH) deficiency and causes hereditary angioedema (HAE). The mechanism of action involves RNA activation (RNAa), specifically boosting hepatic production of C1-INH in HAE patients to prevent recurrent attacks. HAE manifests as episodes of severe swelling in various body parts due to partial C1-INH deficiency[2][3][1].

Targets

Serpin family G member 1 gene promoter

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