viltolarsen   Click here for help

GtoPdb Ligand ID: 11430

Synonyms: NCNP-01 | NS-065 | Viltepso®
Approved drug
viltolarsen is an approved drug (FDA (2020))
Compound class: Nucleic acid
Comment: Viltolarsen is an antisense phosphorodiamidate morpholino oligomer (PMO) class nucleotide drug. Its abbreviated chemical/nucleotide structure is all-P-ambo-[2',3'-azanediyl-P,2',3'-trideoxy-P-(dimethylamino)-2',3'-seco](2'-N→5')(CCTCCGGTTCTGAAGGTGTTC). Viltolarsen restores the reading frame of certain dystrophin gene mutations and promotes synthesis of a shorter but functional dystrophin protein.
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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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were 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/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 141
Hydrogen bond donors 29
Rotatable bonds 122
Topological polar surface area 2504.15
Molecular weight 6921.36
XLogP -21.82
No. Lipinski's rules broken 3

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/)
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 OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CNC[C@@H](O1)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1ncnc2N)n1cnc2c1ncnc2N)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N
Isomeric SMILES OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CNC[C@@H](O1)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1ncnc2N)n1cnc2c1ncnc2N)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N
InChI Key ZVXLKCOOOKREJD-OERAVCCFSA-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 dystrophin (DMD) pre-mRNA
Approved drug? Yes (FDA (2020))
International Nonproprietary Names Click here for help
INN number INN
10771 viltolarsen
Synonyms Click here for help
NCNP-01 | NS-065 | Viltepso®
Database Links Click here for help
CAS Registry No. 2055732-84-6 (source: WHO INN record)
ChEMBL Ligand CHEMBL4298062
DrugBank Ligand DB15005
GtoPdb PubChem SID 440816799
PubChem CID 154562176
Search Google for chemical match using the InChIKey ZVXLKCOOOKREJD-OERAVCCFSA-N
Search Google for chemicals with the same backbone ZVXLKCOOOKREJD
Search PubMed clinical trials viltolarsen
Search PubMed titles viltolarsen
Search PubMed titles/abstracts viltolarsen
UniChem Compound Search for chemical match using the InChIKey ZVXLKCOOOKREJD-OERAVCCFSA-N
UniChem Connectivity Search for chemical match using the InChIKey ZVXLKCOOOKREJD-OERAVCCFSA-N