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revusiran
- Development stage
- Discontinued
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Gene Therapies, 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
Revusiran is a first-generation, chemically synthesized double-stranded small interfering RNA (siRNA) therapeutic designed to target transthyretin (TTR) mRNA in the liver, thereby reducing the production of both wild-type and mutant TTR protein. It is conjugated to a triantennary N-acetylgalactosamine (GalNAc) ligand for targeted delivery to hepatocytes via asialoglycoprotein receptors. Revusiran was developed for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), particularly with cardiomyopathy manifestations. The drug demonstrated potent knockdown of serum TTR levels in clinical studies but was discontinued after phase 3 trials due to an imbalance in mortality observed among treated patients compared with placebo[2][3][5][6].
- Other names
- revusiran
02
Targets
TTR (Transthyretin)
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