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

References
1. Bennett CF, Hung G, Rigo F, Krainer AR, Hua Y, Passini MA, Shihabuddin L, Cheng SH, Klinger KW. (2010)
Compositions and methods for modulation of smn2 splicing in a subject.
Patent number: WO2010148249A1. Assignee: Isis Pharmaceuticals, Inc., Genzyme Corporation, Cold Spring Harbor Laboratory. Priority date: 17/06/2009. Publication date: 23/12/2010.
2. Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, De Vivo DC, Norris DA, Bennett CF, Bishop KM. (2016)
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
Neurology, 86 (10): 890-7. [PMID:26865511]
3. Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E et al.. (2017)
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
Lancet, 388 (10063): 3017-3026. [PMID:27939059]
4. Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E et al.. (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
N. Engl. J. Med., 377 (18): 1723-1732. [PMID:29091570]
5. Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K et al.. (2018)
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.
N. Engl. J. Med., 378 (7): 625-635. [PMID:29443664]
6. Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G et al.. (2011)
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.
Sci Transl Med, 3 (72): 72ra18. [PMID:21368223]
7. Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS et al.. (2014)
Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.
J. Pharmacol. Exp. Ther., 350 (1): 46-55. [PMID:24784568]
8. Rigo F, Hua Y, Chun SJ, Prakash TP, Krainer AR, Bennett CF. (2012)
Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing.
Nat. Chem. Biol., 8 (6): 555-61. [PMID:22504300]