Branaplam hydrochloride (Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride)

Name: Branaplam hydrochloride
Brand: MedChemExpress
SKU: HY-19620A
Rating: 5.0 (15 reviews)

Cat. No.: HY-19620A Purity: 99.43%: Data Sheet
COA

SDS
Handling Instructions
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Technical Support

Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC₅₀ of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC₅₀ of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.

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

CAS No. : 1562338-39-9

Size	Stock	Quantity

Free Sample (0.1 - 0.2 mg)	Apply Now
Solid + Solvent (Highly Recommended)
10 mM * 1 mL in DMSO ready for reconstitution	In-stock	Estimated Time of Arrival: December 31
Solution
10 mM * 1 mL in DMSO	In-stock	Estimated Time of Arrival: December 31
Solid
5 mg	In-stock	Estimated Time of Arrival: December 31
10 mg	In-stock	Estimated Time of Arrival: December 31
25 mg	In-stock	Estimated Time of Arrival: December 31
50 mg	In-stock	Estimated Time of Arrival: December 31
100 mg	In-stock	Estimated Time of Arrival: December 31
200 mg	Get quote
500 mg	Get quote

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Based on 15 publication(s) in Google Scholar

Other Forms of Branaplam hydrochloride:

Branaplam In-stock

15 Publications Citing Use of MCE Branaplam hydrochloride

RT-PCR

Bio/Physico-chemical Assay

Flow Cytometry

Cell Imaging/Staining

: Branaplam hydrochloride purchased from MedChemExpress. Usage Cited in: Cell Rep Methods. 2024 Aug 30:100842. [Abstract]; Western blot for the expression level of OCT4-FLAG in the lysate of cells with or without LMI070 (100 nM).

: Branaplam hydrochloride purchased from MedChemExpress. Usage Cited in: Nature. 2021 Aug;596(7871):291-295. [Abstract]; Exon 7 splicing of the SMN2 and indSMN2 cassette in response to LMI070 or RG7800. Representative RT–PCR reaction showing exon 7 inclusion as a function of LMI070 or RG7800 dose.

: Branaplam hydrochloride purchased from MedChemExpress. Usage Cited in: Nature. 2021 Aug;596(7871):291-295. [Abstract]; Luciferase activity of the SMN2 and indSMN2 cassettes in response to LMI070. The graph shows relative expression of firefly luciferase expressed from the SMN2-on or indSMN2-on cassettes in cells treated with DMSO or LMI070 (100 nM).

: Branaplam hydrochloride purchased from MedChemExpress. Usage Cited in: Nature. 2021 Aug;596(7871):291-295. [Abstract]; Representative FACS analysis of hepatocytes obtained from Ai14 mice after LMI070 (50 mg/kg, i.g.) or vehicle treatment.

: Branaplam hydrochloride purchased from MedChemExpress. Usage Cited in: Nature. 2021 Aug;596(7871):291-295. [Abstract]; Representative photomicrographs of liver sections obtained from AAV injected Ai14 mice 1-week after LMI070 (50 mg/kg, i.g.) treatment.

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Description

In Vitro

Branaplam (LMI070; NVS-SM1) hydrochloride treatment induces changes in the levels of 175 genes in human fibroblasts^[1].

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

In Vivo

Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) hydrochloride produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord^[1].
Branaplam (1 mg/kg of IV; 3 mg/kg of PO) hydrochloride has a CL of 25 mL/min/kg and an AUC of 3.03 μM h^[2].
A single Branaplam (oral; 30 mg/kg) hydrochloride results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice^[1].
Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) hydrochloride improves body weight and extendes lifespan in n SMNΔ7 mice^[1].

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

Clinical Trial

Molecular Weight

429.94

Formula

C₂₂H₂₈ClN₅O₂

CAS No.

1562338-39-9

Appearance

Solid

Color

Light yellow to yellow

SMILES

OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3.Cl

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

4°C, sealed storage, away from moisture and light

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture and light)

Solvent & Solubility

In Vitro:

DMSO : 250 mg/mL (581.48 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)

H₂O : 200 mg/mL (465.18 mM; Need ultrasonic)

Preparing
Stock Solutions

Concentration Solvent Mass	1 mg	5 mg	10 mg

1 mM	2.3259 mL	11.6295 mL	23.2591 mL
5 mM	0.4652 mL	2.3259 mL	4.6518 mL
10 mM	0.2326 mL	1.1630 mL	2.3259 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 (sealed storage, away from moisture and light). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.

Molarity Calculator
Dilution Calculator

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

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

This equation is commonly abbreviated as: C₁V₁ = C₂V₂

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In Vivo Dissolution Calculator

Please enter the basic information of animal experiments:

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Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.

Calculation results:

Working solution concentration: mg/mL

This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.

The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).

Purity & Documentation

Purity: 99.43%

Select Batch:

Data Sheet (274 KB) SDS (252 KB)

COA (249 KB) HNMR (121 KB) LCMS (96 KB)

Handling Instructions (2659 KB)

References

[1]. Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517. [Content Brief]

[2]. Cheung AK, et al. Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Dec 27;61(24):11021-11036. [Content Brief]

Complete Stock Solution Preparation Table

Optional Solvent	Concentration Solvent Mass	1 mg	5 mg	10 mg	25 mg

H₂O / DMSO	1 mM	2.3259 mL	11.6295 mL	23.2591 mL	58.1476 mL
	5 mM	0.4652 mL	2.3259 mL	4.6518 mL	11.6295 mL
	10 mM	0.2326 mL	1.1630 mL	2.3259 mL	5.8148 mL
	15 mM	0.1551 mL	0.7753 mL	1.5506 mL	3.8765 mL
	20 mM	0.1163 mL	0.5815 mL	1.1630 mL	2.9074 mL
	25 mM	0.0930 mL	0.4652 mL	0.9304 mL	2.3259 mL
	30 mM	0.0775 mL	0.3877 mL	0.7753 mL	1.9383 mL
	40 mM	0.0581 mL	0.2907 mL	0.5815 mL	1.4537 mL
	50 mM	0.0465 mL	0.2326 mL	0.4652 mL	1.1630 mL
	60 mM	0.0388 mL	0.1938 mL	0.3877 mL	0.9691 mL
	80 mM	0.0291 mL	0.1454 mL	0.2907 mL	0.7268 mL
	100 mM	0.0233 mL	0.1163 mL	0.2326 mL	0.5815 mL

* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.

Branaplam hydrochloride 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.

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Branaplam hydrochloride (Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride)

Customer Review

Other Forms of Branaplam hydrochloride:

Branaplam hydrochloride Related Antibodies

15 Publications Citing Use of MCE Branaplam hydrochloride

Featured Recommendations

•Related Screening Libraries:

•Related Small Molecules:

•Related Proteins:

View All DNA/RNA Synthesis Isoform Specific Products:

Biological Activity

Purity & Documentation

References

Customer Review

Branaplam hydrochloride Related Antibodies

Molarity Calculator

Dilution Calculator

Complete Stock Solution Preparation Table

Branaplam hydrochloride Related Classifications

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