Givosiran

• CAT Number: I028041
• CAS Number: 1639325-43-1
• Molecular Formula: C78H139N11O30
• Molecular Weight: 1711.017
• Purity: 98%
• Synonyms: Givosiran-linker; Givosiran; ALN-AS1; ALN AS1; ALN-AS 1; WHO 10280; WHO-10280; WHO10280
• 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

Givosiran, also known as ALN-AS1 and WHO 10280, is synthetic small interfering RNA (siRNA) molecule directed against 5-aminolevulinic acid synthase that is used to treat acute hepatic porphyria. Givosiran has been linked to mild-to-moderate ALT elevations during therapy, but has not been linked to instances of idiosyncratic acute liver injury with symptoms and jaundice.