For research use only. Not for therapeutic Use.
Givosiran(CAT: I028041) is a novel small interfering RNA (siRNA) therapeutic that targets aminolevulinic acid synthase 1 (ALAS1), the rate-limiting enzyme in the heme biosynthesis pathway. By silencing ALAS1 mRNA, Givosiran reduces the accumulation of toxic heme precursors, such as aminolevulinic acid (ALA) and porphobilinogen (PBG), which are responsible for the symptoms of acute hepatic porphyria (AHP). Approved for clinical use, Givosiran represents a breakthrough in genetic and metabolic disease research, offering a targeted treatment strategy that addresses the underlying cause of AHP and significantly reduces the frequency of acute attacks and related complications.
| CAS Number | 1639325-43-1 |
| Synonyms | Givosiran-linker; Givosiran; ALN-AS1; ALN AS1; ALN-AS 1; WHO 10280; WHO-10280; WHO10280; |
| Molecular Formula | C78H139N11O30 |
| Purity | 98% |
| Target | Small Interfering RNA (siRNA) |
| Solubility | Soluble in DMSO |
| Appearance | Solid powder |
| Storage | Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years). |
| IUPAC Name | N-[1,3-Bis[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]-12-[(2R,4R)-4-hydroxy-2-methylpyrrolidin-1-yl]-12-oxododecanamide |
| InChI | InChI=1S/C78H139N11O30/c1-50-42-54(96)43-89(50)65(104)26-12-10-8-6-5-7-9-11-25-64(103)88-78(47-111-39-27-61(100)82-33-19-30-79-58(97)22-13-16-36-114-75-66(85-51(2)93)72(108)69(105)55(44-90)117-75,48-112-40-28-62(101)83-34-20-31-80-59(98)23-14-17-37-115-76-67(86-52(3)94)73(109)70(106)56(45-91)118-76)49-113-41-29-63(102)84-35-21-32-81-60(99)24-15-18-38-116-77-68(87-53(4)95)74(110)71(107)57(46-92)119-77/h50,54-57,66-77,90-92,96,105-110H,5-49H2,1-4H3,(H,79,97)(H,80,98)(H,81,99)(H,82,100)(H,83,101)(H,84,102)(H,85,93)(H,86,94)(H,87,95)(H,88,103)/t50-,54-,55-,56-,57-,66-,67-,68-,69+,70+,71+,72-,73-,74-,75-,76-,77-/m1/s1 |
| InChIKey | RUPXJRIDSUCQAN-PQNNUJSWSA-N |
| SMILES | O=C(NC(COCCC(NCCCNC(CCCCO[C@@H]1O[C@H](CO)[C@H](O)[C@H](O)[C@H]1NC(C)=O)=O)=O)(COCCC(NCCCNC(CCCCO[C@@H]2O[C@H](CO)[C@H](O)[C@H](O)[C@H]2NC(C)=O)=O)=O)COCCC(NCCCNC(CCCCO[C@@H]3O[C@H](CO)[C@H](O)[C@H](O)[C@H]3NC(C)=O)=O)=O)CCCCCCCCCCC(N4[C@H](C)C[C@@H](O)C4)=O |
