Givosiran | CAS#1639325-43-1

MedKoo Cat#: 533657 | Name: Givosiran

Description:

WARNING: This product is for research use only, not for human or veterinary use.

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.

Chemical Structure

Givosiran

CAS#1639325-43-1

Theoretical Analysis

MedKoo Cat#: 533657

Name: Givosiran

CAS#: 1639325-43-1

Chemical Formula: C78H139N11O30

Exact Mass: 1709.9689

Molecular Weight: 1711.02

Elemental Analysis: C, 54.75; H, 8.19; N, 9.00; O, 28.05

Price and Availability

This product is currently not in stock but may be available through custom synthesis. To ensure cost efficiency, the minimum order quantity is 1 gram. The estimated lead time is 2 to 4 months, with pricing dependent on the complexity of the synthesis (typically high for intricate chemistries). Quotes for quantities below 1 gram will not be provided. To request a quote, please click the button below. Note: If this product becomes available in stock in the future, pricing will be listed accordingly.

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Related CAS #

1469730-77-5 (hydroxy)

Synonym

Givosiran-linker; Givosiran; ALN-AS1; ALN AS1; ALN-AS 1; WHO 10280; WHO-10280; WHO10280;

IUPAC/Chemical 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 Key

RUPXJRIDSUCQAN-PQNNUJSWSA-N

InChi Code

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

SMILES Code

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

Appearance

Solid powder

Purity

>98% (or refer to the Certificate of Analysis)

Shipping Condition

Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition

Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility

Soluble in DMSO

Shelf Life

>3 years if stored properly

Drug Formulation

This drug may be formulated in DMSO

Stock Solution Storage

0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code

2934.99.9001

More Info

ESC-GalNAc-siRNA conjugate ALN-AS1 RNA interference therapeutic ALN-AS1 RNAi ALN-AS1 Givosiran is a proprietary enhanced stabilization chemistry (ESC)-stabilized conjugate composed of the liver-targeted ligand N-acetylgalactosamine (GalNAc) conjugated to small-interfering RNAs (siRNAs) directed against the liver-expressed enzyme aminolevulinic acid synthase 1 (delta-aminolevulinate synthase 1; ALAS1; ALAS-1) that can potentially be used in the treatment of acute hepatic porphyrias (AHPs). Upon subcutaneous administration of givosiran, the GalNAc moiety targets and binds with high affinity to asialoglycoprotein receptors (ASGPRs) expressed on hepatocytes. Once inside the cell, the siRNAs bind to and silence ALAS1 mRNA and inhibit both the translation and expression of the ALAS1 protein. This prevents delta-aminolevulinic acid (ALA) formation, decreases 5-ALA levels and prevents the production of porphyrins and hemes, such as porphobilinogen (PBG). AHPs are a group of metabolic disorders caused by deficiencies of specific enzymes that are responsible for hemoglobulin biosynthesis within the liver, which leads to the accumulation of toxic intermediates, such as ALA and PBG. ALAS1, a liver-expressed, rate-limiting enzyme in the heme biosynthesis pathway, is responsible for the formation of ALA from succinyl-CoA and glycine. ESC enables the subcutaneous dosing of givosiran with increased efficacy, durability and a wide therapeutic index as compared to non-ESC GalNAc-siRNA conjugates. (https://www.cancer.gov/publications/dictionaries/cancer-drug/def/givosiran)

Instruction

View handling instruction

Preparing Stock Solutions

The following data is based on the product molecular weight 1,711.02 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.