nusinersen

Ligand id: 9416

Name: nusinersen

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 189
Hydrogen bond donors 27
Rotatable bonds 176
Topological polar surface area 3045.87
Molecular weight 7126.31
XLogP -31.15
No. Lipinski's rules broken 3

Molecular properties generated using the CDK

Classification
Compound class Synthetic organic
Approved drug? Yes (source: FDA (2016))
International Nonproprietary Names
INN number INN
10003 nusinersen
Synonyms
ASO-10-27
Ionis-SMNrx
ISIS 396443
ISIS-SMNRx
ISIS396443
Spinraza®
Database Links
CAS Registry No. 1258984-36-9 (source: WHO INN record)
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
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Search PubMed titles/abstracts nusinersen
Comments
Nusinersen (ISIS 396443) is an antisense oligo(ribo)nucleotide (ASO) which induces survival of motor neuron (SMN) expression [5].
Nusinersen is designated as an orphan drug by the US FDA and EMA and is the first drug 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].