For research use only. Not for therapeutic Use.
Cemdisiran is an N-acetylgalactosamine (GalNAc) conjugated siRNA for the research of complement-mediated diseases by suppressing liver production of complement 5 (C5) protein.
| CAS Number | 1639264-46-2 |
| Synonyms | [(2S,4R)-1-[12-[[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]amino]-12-oxododecanoyl]-4-hydroxypyrrolidin-2-yl]methyl dihydrogen phosphate |
| Molecular Formula | C542H711F17N169O330P45S6 |
| Purity | ≥95% |
| InChI | InChI=1S/C78H140N11O34P/c1-50(93)85-66-72(108)69(105)55(43-90)121-75(66)117-35-15-12-21-58(97)79-29-18-32-82-61(100)26-38-114-47-78(88-64(103)24-10-8-6-4-5-7-9-11-25-65(104)89-42-54(96)41-53(89)46-120-124(111,112)113,48-115-39-27-62(101)83-33-19-30-80-59(98)22-13-16-36-118-76-67(86-51(2)94)73(109)70(106)56(44-91)122-76)49-116-40-28-63(102)84-34-20-31-81-60(99)23-14-17-37-119-77-68(87-52(3)95)74(110)71(107)57(45-92)123-77/h53-57,66-77,90-92,96,105-110H,4-49H2,1-3H3,(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)(H2,111,112,113)/t53-,54+,55+,56+,57+,66+,67+,68+,69-,70-,71-,72+,73+,74+,75+,76+,77+/m0/s1 |
| InChIKey | LQRNAUZEMLGYOX-LZVIIAQDSA-N |
| SMILES | CC(=O)NC1C(C(C(OC1OCCCCC(=O)NCCCNC(=O)CCOCC(COCCC(=O)NCCCNC(=O)CCCCOC2C(C(C(C(O2)CO)O)O)NC(=O)C)(COCCC(=O)NCCCNC(=O)CCCCOC3C(C(C(C(O3)CO)O)O)NC(=O)C)NC(=O)CCCCCCCCCCC(=O)N4CC(CC4COP(=O)(O)O)O)CO)O)O |
| Reference | [1]. Prajakta Badri, et al. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021;60(3):365-378. [2]. Badri P, Jiang X, Borodovsky A, et al. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021;60(3):365-378. |
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