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Vutrisiran

Cat. No. M40955
Vutrisiran Structure
Synonym:

ALN-TTRsc02

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Biological Activity

Vutrisiran is a liver-directed small interfering RNA (siRNA) that targets transthyretin protein (TTR) mRNA and can be used in TTR-mediated amyloidosis studies.

Chemical Information
Molecular Weight 16345
CAS Number 1867157-35-4
Form Solid
Storage 4°C, dry, sealed
Conversion of different model animals based on BSA (PMID: 27057123)
Species Mouse Rat Rabbit Guinea pig Hamster Dog
Weight (kg) 0.02 0.15 1.8 0.4 0.08 10
Body Surface Area (m2) 0.007 0.025 0.15 0.05 0.02 0.5
Km factor 3 6 12 8 5 20
Animal A (mg/kg) = Animal B (mg/kg) multiplied by  Animal B Km
Animal A Km

For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.

References

[1] Susan J Keam. Drugs. Vutrisiran: First Approval

[2] No authors listed. Am J Health Syst Pharm. Vutrisiran Sodium

[3] David Adams et al. Amyloid. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

[4] Bahru A Habtemariam et al. Clin Pharmacol Ther. Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

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Keywords: Vutrisiran, ALN-TTRsc02 supplier, Transthyretin (TTR), inhibitors, activators


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