Medals oligonucleic drug ID monrd0029
ATC Classification -
Product Name Nusinersen
Synonims Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Country US
Company Ionis Pharmaceuticals
BioMarin Nederland BV
phase AD
Type Antisense
Adaptation Diseases Infantile-onset Spinal Muscular Atrophy
Target Molecule Survival of Motor Neuron 2 (SMN2) Splicing Modulator
Remarks Nusinersen is a drug under development for the treatment of spinal muscular atrophy.
In an open-label phase II clinical dose clinical trial, safety, tolerability, pharmacokinetics and clinical efficacy of multiple intrathecal administration to infantile onset spinal muscular atrophy patients were evaluated.
Since both items were satisfied, a phase III trial has been carried out.
Nucleic Acid Sequence 5’-mU-mC-A-mC-mU-mU-mU-mC-A-mU-A-A-mU-G-mC-mU-G-G-3’
DrugBank -
TargetProtein - UniProt Q16637
TargetProtein - PDB 2LEH