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

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 Price Stock Quantity
Solid + Solvent (Highly Recommended)
10 mM * 1 mL in DMSO
ready for reconstitution
 USD 638 In-stock
Solution
10 mM * 1 mL in DMSO USD 638 In-stock
Solid
1 mg USD 232 In-stock
5 mg USD 580 In-stock
10 mg USD 940 In-stock
25 mg USD 1900 In-stock
50 mg USD 3050 In-stock
100 mg USD 4800 In-stock
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Based on 1 publication(s) in Google Scholar

NVS-SM2 Related Antibodies

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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].

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

NVS-SM2 Related Antibodies
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 NVS-SM2 treatment induces a 1.5-fold increase in SMN protein levels in the mouse brain[1].

MedChemExpress (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.
Appearance

Solid

Color

Off-white to light yellow

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
Powder -20°C 3 years
In solvent -80°C 6 months
-20°C 1 month
Solvent & Solubility
In Vitro: 

DMSO : 75 mg/mL (184.49 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.4599 mL 12.2995 mL 24.5990 mL
5 mM 0.4920 mL 2.4599 mL 4.9198 mL
View the Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

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

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

This equation is commonly abbreviated as: C1V1 = C2V2

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In Vivo Dissolution Calculator
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Calculation results:
Working solution concentration: mg/mL
Purity & Documentation
References

Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

Optional Solvent Concentration Solvent Mass 1 mg 5 mg 10 mg 25 mg
DMSO 1 mM 2.4599 mL 12.2995 mL 24.5990 mL 61.4976 mL
5 mM 0.4920 mL 2.4599 mL 4.9198 mL 12.2995 mL
10 mM 0.2460 mL 1.2300 mL 2.4599 mL 6.1498 mL
15 mM 0.1640 mL 0.8200 mL 1.6399 mL 4.0998 mL
20 mM 0.1230 mL 0.6150 mL 1.2300 mL 3.0749 mL
25 mM 0.0984 mL 0.4920 mL 0.9840 mL 2.4599 mL
30 mM 0.0820 mL 0.4100 mL 0.8200 mL 2.0499 mL
40 mM 0.0615 mL 0.3075 mL 0.6150 mL 1.5374 mL
50 mM 0.0492 mL 0.2460 mL 0.4920 mL 1.2300 mL
60 mM 0.0410 mL 0.2050 mL 0.4100 mL 1.0250 mL
80 mM 0.0307 mL 0.1537 mL 0.3075 mL 0.7687 mL
100 mM 0.0246 mL 0.1230 mL 0.2460 mL 0.6150 mL
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NVS-SM2 Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

NVS-SM21562333-92-9DNA/RNA Synthesissurvivalmotorneuronspinalmuscularatrophypre-mRNAsplicingSMN2brain-penetrantInhibitorinhibitorinhibit

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