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rapirosiran
- Development stage
- Phase 2
- Lead developer
- Regeneron Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Rapirosiran is an investigational N-acetylgalactosamine (GalNAc)-conjugated small interfering RNA (siRNA) therapeutic designed to silence the HSD17B13 gene, which encodes hydroxysteroid 17-beta dehydrogenase 13, a liver-expressed protein implicated in the pathogenesis of non-alcoholic steatohepatitis (NASH) and metabolic dysfunction-associated steatohepatitis (MASH). By targeting and reducing HSD17B13 mRNA, rapirosiran aims to lower hepatic expression of the protein and mitigate liver injury and fibrosis associated with these conditions. Rapirosiran is being developed by Alnylam Pharmaceuticals and Regeneron Pharmaceuticals. Clinical trials have shown rapirosiran induces robust, dose-dependent knockdown of hepatic HSD17B13 mRNA and has a favorable safety and tolerability profile in both healthy volunteers and patients with MASH/NASH[1][2][3][6][8].
- Other names
- rapirosiran sodium
02
Targets
HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)
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