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vutrisiran
- Development stage
- Approved
- Lead developer
- Alnylam 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
Vutrisiran is a double-stranded small interfering RNA (siRNA) therapeutic used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. It works by targeting and degrading both mutant and wild-type transthyretin (TTR) messenger RNA in the liver, thereby reducing the production of TTR protein. This reduction leads to fewer amyloid deposits in tissues, addressing nerve damage and heart complications associated with these conditions. Vutrisiran utilizes a GalNAc conjugate delivery platform for targeted uptake by hepatocytes, allowing for subcutaneous administration at extended intervals. The drug was developed by Alnylam Pharmaceuticals and is marketed under the brand name Amvuttra[1][4][5][6].
- Brand names
- Amvuttra
- Other names
- vutrisiranALN-TTRsc02ALN-TTRsc-02ALN-TTRsc 02
02
Targets
TTR (Transthyretin)
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