1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis
  3. NVS-SM2

NVS-SM2 

Cat. No.: HY-111520
Handling Instructions

NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research.

For research use only. We do not sell to patients.

NVS-SM2 Chemical Structure

NVS-SM2 Chemical Structure

CAS No. : 1562333-92-9

Size Stock
100 mg   Get quote  
250 mg   Get quote  
500 mg   Get quote  

* Please select Quantity before adding items.

Top Publications Citing Use of Products
  • Biological Activity

  • Purity & Documentation

  • References

  • Customer Review

Description

NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research[1][2].

IC50 & Target

EC50: 5 nM (SMN)[1]

In Vitro

For NVS-SM2, the molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5' splice site is increased in a sequence-selective manner, discrete from constitutive recognition[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model[2].
Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with Tmax of 3 h after PO with 3 mg/kg in mice, and treatment induced a 1.5-fold increase in SMN protein levels in the mouse brain[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Severe SMA mice[2]
Dosage: 0.1 mg/kg and 1 mg/kg
Administration: Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30
Result: Extended survival in a severe SMA mouse model.
Molecular Weight

406.52

Formula

C₂₃H₃₀N₆O

CAS No.
SMILES

OC1=C(C(N=N2)=CC=C2N(C(CC3(C)C)CC(C)(N3)C)C)C=CC(C4=CNN=C4)=C1

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

Please store the product under the recommended conditions in the Certificate of Analysis.

References
  • No file chosen (Maximum size is: 1024 Kb)
  • If you have published this work, please enter the PubMed ID.
  • Your name will appear on the site.
  • Molarity Calculator

  • Dilution Calculator

The molarity calculator equation

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass   Concentration   Volume   Molecular Weight *
= × ×

The dilution calculator equation

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
× = ×
C1   V1   C2   V2

Keywords:

NVS-SM2DNA/RNA Synthesissurvivalmotorneuronspinalmuscularatrophypre-mRNAsplicingSMN2brain-penetrantInhibitorinhibitorinhibit

Your Recently Viewed Products:

Inquiry Online

Your information is safe with us. * Required Fields.

Product Name

 

Salutation

Applicant Name *

 

Email address *

Phone number *

 

Organization name *

Department *

 

Requested quantity *

Country or Region *

     

Remarks

Bulk Inquiry

Inquiry Information

Product Name:
NVS-SM2
Cat. No.:
HY-111520
Quantity:
MCE Japan Authorized Agent: