nusinersen

Ligand id: 9416

Name: nusinersen

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 161
Hydrogen bond donors 23
Rotatable bonds 176
Topological polar surface area 3144.17
Molecular weight 7122.28
XLogP -19.23
No. Lipinski's rules broken 3

Molecular properties generated using the CDK

Classification
Compound class Synthetic organic
Approved drug? Yes (FDA (2016), EMA (2017))
International Nonproprietary Names
INN number INN
10003 nusinersen
Synonyms
ASO-10-27 | Ionis-SMNrx | ISIS 396443 | ISIS-SMNRx | ISIS396443 | Spinraza®
Comments
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. A full structural rendering using SMILES from Chemspider is available here (CSID:34983394). The sequence for nusinersen is claimed in patent WO2010148249A1 as SEQ ID NO: 1 [1].
Database Links
CAS Registry No. 1258984-36-9 (source: WHO INN record)
GtoPdb PubChem SID 328083518
PubChem CID 124037382
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
Search UniChem for chemical match using the InChIKey WWFDJIVIDXJAQR-FFWSQMGZSA-N
Search UniChem for chemicals with the same backbone WWFDJIVIDXJAQR