Bioactivity | Givosiran sodium is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran sodium downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran sodium can be used for the research of acute intermittent porphyria[1][2]. |
Formula | C524H650F16N173Na44O316P43S6 |
Molar Mass | 17267.50 |
Appearance | 固体 |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | -20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
Reference | [1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339. [2]. Balwani M, et, al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301. |