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nusinersen   Click here for help

GtoPdb Ligand ID: 9416

Synonyms: ASO-10-27 | BIIB-058 | BIIB058 | Ionis-SMNrx | ISIS 396443 | ISIS SMNRx | ISIS-SMNRx | ISIS396443 | Spinraza®
Approved drug
nusinersen is an approved drug (FDA (2016), EMA (2017))
Compound class: Nucleic acid
Comment: Nusinersen (ISIS 396443) is an antisense oligo(ribo)nucleotide (ASO) which induces survival motor neuron (SMN) protein expression from SMN2 genes with the exon 7-skipping mutation [7].
Nusinersen was initially available for use as a designated orphan drug (by the US FDA and EMA), and was the first drug to be fully approved for the treatment of spinal muscular atrophy (SMA).

The sequence contains 2'-O-(2-methoxyethyl) (2'-MOE)-oligoribonucleotides to reduce nuclease degradation and enhance binding affinity towards the complementary RNA. The full sequence is [2'-O-(2-methoxyethyl)](3'-5')(P-thio)(mU-mC-A-mC-mU-mU-mU-mC-A-mU-A-A-mU-G-mC-mU-G-G) as detailed in the INN record for the agent.
The sequence for nusinersen is claimed in patent WO2010148249A1 as SEQ ID NO: 1 [1].
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES COCCOC1C(OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2OP(=O)(OCC2OC(C(C2O)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)OCCOC)n2cc(C)c(nc2=O)N)S)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)OCCOC)n2cnc3c2ncnc3N)S)OCCOC)n2cnc3c2ncnc3N)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)C(OC1n1cnc2c1ncnc2N)COP(=O)(OC1C(COP(=O)(OC2C(COP(=O)(OC3C(COP(=O)(OC4C(COP(=O)(OC5C(COP(=O)(OC6C(COP(=O)(OC7C(COP(=O)(OC8C(CO)OC(C8OCCOC)n8cc(C)c(=O)[nH]c8=O)S)OC(C7OCCOC)n7cc(C)c(nc7=O)N)S)OC(C6OCCOC)n6cnc7c6ncnc7N)S)OC(C5OCCOC)n5cc(C)c(nc5=O)N)S)OC(C4OCCOC)n4cc(C)c(=O)[nH]c4=O)S)OC(C3OCCOC)n3cc(C)c(=O)[nH]c3=O)S)OC(C2OCCOC)n2cc(C)c(=O)[nH]c2=O)S)OC(C1OCCOC)n1cc(C)c(nc1=O)N)S
Isomeric SMILES COCCO[C@@H]1[C@H](OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2OP(=O)(OC[C@H]2O[C@H]([C@@H]([C@@H]2O)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)OCCOC)n2cc(C)c(nc2=O)N)S)OCCOC)n2cnc3c2nc(N)[nH]c3=O)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)OCCOC)n2cnc3c2ncnc3N)S)OCCOC)n2cnc3c2ncnc3N)S)OCCOC)n2cc(C)c(=O)[nH]c2=O)S)[C@H](O[C@H]1n1cnc2c1ncnc2N)COP(=O)(O[C@@H]1[C@@H](COP(=O)(O[C@@H]2[C@@H](COP(=O)(O[C@@H]3[C@@H](COP(=O)(O[C@@H]4[C@@H](COP(=O)(O[C@@H]5[C@@H](COP(=O)(O[C@@H]6[C@@H](COP(=O)(O[C@@H]7[C@@H](COP(=O)(O[C@@H]8[C@@H](CO)O[C@H]([C@@H]8OCCOC)n8cc(C)c(=O)[nH]c8=O)S)O[C@H]([C@@H]7OCCOC)n7cc(C)c(nc7=O)N)S)O[C@H]([C@@H]6OCCOC)n6cnc7c6ncnc7N)S)O[C@H]([C@@H]5OCCOC)n5cc(C)c(nc5=O)N)S)O[C@H]([C@@H]4OCCOC)n4cc(C)c(=O)[nH]c4=O)S)O[C@H]([C@@H]3OCCOC)n3cc(C)c(=O)[nH]c3=O)S)O[C@H]([C@@H]2OCCOC)n2cc(C)c(=O)[nH]c2=O)S)O[C@H]([C@@H]1OCCOC)n1cc(C)c(nc1=O)N)S
InChI Key WWFDJIVIDXJAQR-FFWSQMGZSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
Classification Click here for help
Compound class Nucleic acid
Compound subclass Antisense oligonucleotide (ASO)
Target survival of motor neuron 2, centromeric (SMN2) with exon 7 skipping mutations
Approved drug? Yes. EU EMA (2017)  |  US FDA (2016)
IUPAC Name Click here for help
O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-cytidylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-adenylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-cytidylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-cytidylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-adenylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-adenylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-adenylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-guanylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-cytidylyl-(3'->5')-O2'-(2-methoxyethyl)-5-methyl-P-thio-uridylyl-(3'->5')-O2'-(2-methoxyethyl)-P-thio-guanylyl-(3'->5')-O2'-(2-methoxyethyl)-guanosine
International Nonproprietary Names Click here for help
INN number INN
10003 nusinersen
Synonyms Click here for help
ASO-10-27 | BIIB-058 | BIIB058 | Ionis-SMNrx | ISIS 396443 | ISIS SMNRx | ISIS-SMNRx | ISIS396443 | Spinraza®
Database Links Click here for help
CAS Registry No. 1258984-36-9 (source: WHO INN record)
ChEMBL Ligand CHEMBL3301572
DrugBank Ligand DB13161
GtoPdb PubChem SID 328083518
PubChem CID 131801471
Search Google for chemical match using the InChIKey WWFDJIVIDXJAQR-FFWSQMGZSA-N
Search Google for chemicals with the same backbone WWFDJIVIDXJAQR
Search PubMed clinical trials nusinersen
Search PubMed titles nusinersen
Search PubMed titles/abstracts nusinersen
UniChem Compound Search for chemical match using the InChIKey WWFDJIVIDXJAQR-FFWSQMGZSA-N
UniChem Connectivity Search for chemical match using the InChIKey WWFDJIVIDXJAQR-FFWSQMGZSA-N