Branaplam
Based on 16 publication(s) in Google Scholar
Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.
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- Pureza: 98.90%
- No. CAS: 1562338-42-4
- Fòrmula: C22H27N5O2
- Peso molecular:393.48
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Almacenamiento:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 1 year; -20°C, 6 months (sealed storage, away from moisture)
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Actividad biológica
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Chemical Information
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Solvente y solubilidad
- Pureza y Documentación
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Publications Citing Use of MedChemExpress (MCE) Branaplam
More- Nature. 2021 Aug;596(7871):291-295. [Abstract]
- Nat Commun. 2026 May 30. [Abstract]
- Nucleic Acids Res. 2023 Jul 7;51(12):5948-5980. [Abstract]
- Nucleic Acids Res. 2021 Sep 7;49(15):8462-8470. [Abstract]
- Mol Ther Nucleic Acids. 2024 Mar 11;35(2):102172. [Abstract]
- Cell Rep Methods. 2024 Aug 30:100842. [Abstract]
- Neurosci Insights. 2024 Feb 19:19:26331055241233596. [Abstract]
- bioRxiv. 2025 Dec 2.
- bioRxiv. 2025 Nov 24:2025.10.17.683097. [Abstract]
- bioRxiv. 2025 May 28:2025.05.28.656493. [Abstract]
- University of Basel. 2024
- bioRxiv. 2024 Apr 18.
- Patent. US20230340498A1.
- bioRxiv. 2023 Jul 27:2023.07.25.550489. [Abstract]
- bioRxiv. 2020 Jun.
- bioRxiv. 2020 Feb.
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Actividad biológica
Branaplam (LMI070; NVS-SM1) 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.
? Branaplam (1 mg/kg of IV; 3 mg/kg of PO) has a CL of 25 mL/min/kg and an AUC of 3.03 μM?h[2].
? A single Branaplam (oral; 30 mg/kg) 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) 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.
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Animal Model:C/+ SMA mouse model[1]
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Dosage:3, 10, 30 mg/kg
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Administration:Oral
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Result:Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord.
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Animal Model:Male Sprague-Dawley rat[2]
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Dosage:1 mg/kg (IV);3 mg/kg (PO) (Pharmacokinetic Analysis)
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Administration:IV or PO
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Result:Had a CL of 25 mL/min/kg and an AUC of 3.03 μM•h.
Chemical Information
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No. CAS 1562338-42-4
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Appearance Solid
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Peso molecular 393.48
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Fòrmula C22H27N5O2
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Color White to yellow
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SMILES
OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3
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Synonyms
LMI070; NVS-SM1
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
4°C, sealed storage, away from moisture
* In solvent : -80°C, 1 year; -20°C, 6 months (sealed storage, away from moisture)
Publications (16)
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Journal Impact Factor
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Most Recent
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Nature
2021 Aug;596(7871):291-295. PMID: 34321659
Branaplam 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 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 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 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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Nat Commun
2026 May 30. PMID: 42218123 -
Nucleic Acids Res
Diverse targets of SMN2-directed splicing-modulating small molecule therapeutics for spinal muscular atrophy. [Abstract]2023 Jul 7;51(12):5948-5980. PMID: 37026480 -
Nucleic Acids Res
A small-molecule fluorescence probe ANP77 for sensing RNA internal loop of C, U and A/CC motifs and their binding molecules. [Abstract]2021 Sep 7;49(15):8462-8470. PMID: 34358308 -
Mol Ther Nucleic Acids
Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity. [Abstract]2024 Mar 11;35(2):102172. PMID: 38978694 -
Cell Rep Methods
A compact, versatile drug-induced splicing switch system with minimal background expression. [Abstract]2024 Aug 30:100842. PMID: 39236714
Branaplam 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).
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Neurosci Insights
Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene. [Abstract]2024 Feb 19:19:26331055241233596. PMID: 38379891 -
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bioRxiv
Systematic characterization of existing and novel inducible transgenic systems in human pluripotent stem cells after prolonged differentiation. [Abstract]2025 Nov 24:2025.10.17.683097. PMID: 41311869 -
bioRxiv
2025 May 28:2025.05.28.656493. PMID: 40501903 -
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bioRxiv
PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease. [Abstract]2023 Jul 27:2023.07.25.550489. PMID: 37547003 -
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Solvente y solubilidad
DMSO : 3.33 mg/mL (8.46 mM; ultrasonic and warming and heat to 60°C; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)
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, 1 year; -20°C, 6 months (sealed storage, away from moisture). When stored at -80°C, please use it within 1 year. When stored at -20°C, please use it within 6 months.
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, 1 year; -20°C, 6 months (sealed storage, away from moisture). When stored at -80°C, please use it within 1 year. When stored at -20°C, please use it within 6 months.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Select the appropriate dissolution method based on your experimental animal and administration route.
- For the following dissolution methods, please ensure to first prepare a clear stock solution using an In Vitro approach and then sequentially add co-solvents:
- To ensure reliable experimental results, the clarified stock solution can be appropriately stored based on storage conditions. As for the working solution for In Vivo experiments, it is recommended to prepare freshly and use it on the same day.
- The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: 10% DMSO 40% PEG300 5% Tween-80 45% Saline
Solubility: ≥ 0.33 mg/mL (0.84 mM); Clear solution
This protocol yields a clear solution of ≥ 0.33 mg/mL (saturation unknown).
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (3.3 mg/mL) to 400 μL PEG300, and mix evenly; then add 50 μL Tween-80 and mix evenly; then add 450 μL Saline to adjust the volume to 1 mL.
Preparation of Saline: Dissolve 0.9 g sodium chloride in ddH₂O and dilute to 100 mL to obtain a clear Saline solution.
Add each solvent one by one: 10% DMSO 90% (20% SBE-β-CD in Saline)
Solubility: ≥ 0.33 mg/mL (0.84 mM); Clear solution
This protocol yields a clear solution of ≥ 0.33 mg/mL (saturation unknown).
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (3.3 mg/mL) to 900 μL 20% SBE-β-CD in Saline, and mix evenly.
Preparation of 20% SBE-β-CD in Saline (4°C, storage for one week): 2 g SBE-β-CD powder is dissolved in 10 mL Saline, completely dissolve until clear.
Add each solvent one by one: 1% DMSO 99% Saline
Solubility: 0.12 mg/mL (0.30 mM); Suspended solution; Need ultrasonic
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: 50% PEG300 50% Saline
Solubility: 5 mg/mL (12.71 mM); Suspended solution; Need ultrasonic
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.
Please enter your animal formula composition:
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%DMSO +
Recommended: Keep the proportion of DMSO in working solution below 2% if your animal is weak.
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%+
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+%Tween-80 + +
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%Saline +
The co-solvents required include: DMSO, . All of co-solvents are available by MedChemExpress (MCE). , Tween 80. All of co-solvents are available by MedChemExpress (MCE).
Working solution concentration: 0.22 mg/mL
Method for preparing stock solution: mg drug dissolved in μL DMSO. Stock solution concentration: mg/mL. * In solvent : -80°C, 1 year; -20°C, 6 months (sealed storage, away from moisture)
1. Take μL DMSO stock solution;
2. Add μL .
μL , mix evenly;
3. Then add μL Tween 80, mix evenly;
4. Then add μL
Please ensure that the stock solution in the first step is dissolved to a clear state, and add co-solvents in sequence. You can use ultrasonic heating (ultrasonic cleaner, recommended frequency 20-40 kHz), vortexing, etc. to assist dissolution.
Pureza y Documentación
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Ficha de datos (277 KB)
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SDS (393 KB)
- English - EN (393 KB)
- Français - FR (393 KB)
- Deutsch - DE (393 KB)
- Norwegian - NO (393 KB)
- Español - ES (393 KB)
- Swedish - SV (393 KB)
- Italian - IT (393 KB)
- Korean - KR (393 KB)
- Portuguese - PT (393 KB)
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Instrucciones de manejo (2659 KB)
Referencias
[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
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, 1 year; -20°C, 6 months (sealed storage, away from moisture). When stored at -80°C, please use it within 1 year. When stored at -20°C, please use it within 6 months.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| DMSO | 1 mM | 2.5414 mL | 12.7071 mL | 25.4143 mL | 63.5356 mL |
| 5 mM | 0.5083 mL | 2.5414 mL | 5.0829 mL | 12.7071 mL |
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