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Branaplam hydrochloride

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Catalog No. T62381Cas No. 1562338-39-9
Alias NVS-SM1 hydrochloride, LMI070 hydrochloride, LMI 070 hydrochloride

Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.

Branaplam hydrochloride

Branaplam hydrochloride

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Purity: 98.45%
Catalog No. T62381Alias NVS-SM1 hydrochloride, LMI070 hydrochloride, LMI 070 hydrochlorideCas No. 1562338-39-9
Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.
Pack SizePriceUSA WarehouseGlobal WarehouseQuantity
1 mg$39-In Stock
5 mg$83-In Stock
10 mg$118-In Stock
25 mg$198-In Stock
50 mg$293-In Stock
100 mg$433-In Stock
200 mg$642-In Stock
1 mL x 10 mM (in DMSO)$98-In Stock
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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]
All TargetMol products are for research purposes only and cannot be used for human consumption. We do not provide products or services to individuals. Please comply with the intended use and do not use TargetMol products for any other purpose.
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Purity:98.45%
Appearance:Solid
Color:Yellow
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Product Introduction

Bioactivity
Description
Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.
Targets&IC50
SMN:20 nM (EC50), ERG (human):6.3 μM
In vitro
In Homo sapiens fibroblasts, Branaplam hydrochloride caused changes in the expression levels of 175 genes. [1]
In vivo
After oral administration of Branaplam hydrochloride (3, 10, 30 mg/kg), the SMN2-FL transcript and SMN protein levels in the brain and spinal cord of mice showed a dose-dependent increase [1].
SynonymsNVS-SM1 hydrochloride, LMI070 hydrochloride, LMI 070 hydrochloride
Chemical Properties
Molecular Weight429.95
FormulaC22H28ClN5O2
Cas No.1562338-39-9
SmilesCl.OC1=CC(=CC=C1C=2N=NC(OC3CC(NC(C)(C)C3)(C)C)=CC2)C=4C=NNC4
Storage & Solubility Information
Storagekeep away from moisture,keep away from direct sunlight | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.
Solubility Information
H2O: <1mg/mL (Insoluble)
DMSO: 200 mg/mL (465.17 mM), Sonication is recommended.
Solution Preparation Table
DMSO
1mg5mg10mg50mg
1 mM2.3259 mL11.6293 mL23.2585 mL116.2926 mL
5 mM0.4652 mL2.3259 mL4.6517 mL23.2585 mL
10 mM0.2326 mL1.1629 mL2.3259 mL11.6293 mL
20 mM0.1163 mL0.5815 mL1.1629 mL5.8146 mL
50 mM0.0465 mL0.2326 mL0.4652 mL2.3259 mL
100 mM0.0233 mL0.1163 mL0.2326 mL1.1629 mL

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Please enter your animal experiment information in the following box and click Calculate to obtain the stock solution preparation method and in vivo formula preparation method:
TargetMol | Animal experiments For example, if the intended dosage is 10 mg/kg for animals weighing 20 g , with a dosing volume of 100 μL per animal, TargetMol | Animal experiments and a total of 10 animals are to be administered, using a formulation of TargetMol | reagent 10% DMSO+ 40% PEG300+ 5% Tween 80+ 45% Saline/PBS/ddH2O , the resulting working solution concentration would be 2 mg/mL.
Stock Solution Preparation:

Dissolve 2 mg of the compound in 100 μL DMSOTargetMol | reagent to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.

Preparation of the In Vivo Formulation:

1) Add 100 μL of the DMSOTargetMol | reagent stock solution to 400 μL PEG300TargetMol | reagent and mix thoroughly until the solution becomes clear.

2) Add 50 μL Tween 80 and mix well until fully clarified.

3) Add 450 μL Saline,PBS or ddH2OTargetMol | reagent and mix thoroughly until a homogeneous solution is obtained.

This example is provided solely to demonstrate the use of the In Vivo Formulation Calculator and does not constitute a recommended formulation for any specific compound. Please select an appropriate dissolution and formulation strategy based on your experimental model and route of administration.
All co-solvents required for this protocol, includingDMSO, PEG300/PEG400, Tween 80, SBE-β-CD, and Corn oil, are available for purchase on the TargetMol website.
1 Enter information below:
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2 Enter the in vivo formulation:
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