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

Bioactivity Comments
The molecular defect of this disease derives from a C-to-T transition in an exonic splicing enhancer site which impairs the ability of the spliceosome to recognize exon 7. The molecular mechanism of action of this drug is blocking the intronic splicing silencer of the 5′ region of SMN2 intron 7 (ISS-N1). It thereby promotes exon 7 inclusion and a corresponding increase in full-length SMN2 protein [4].