Branaplam hydrochloride
Based on 16 publication(s) in Google Scholar
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.
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- Pureté: 99.43%
- CAS No.: 1562338-39-9
- Formule: C22H28ClN5O2
- Masse moléculaire:429.94
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Stockage:
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)
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Activité biologique
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Chemical Information
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Solvant et solubilité
- Pureté et documentation
- Références
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Publications Citing Use of MedChemExpress (MCE) Branaplam hydrochloride
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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Activité biologique
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.
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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 1562338-39-9
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Appearance Solid
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Masse moléculaire 429.94
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Formule C22H28ClN5O2
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Color Light yellow 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.Cl
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Synonyms
LMI070 hydrochloride; NVS-SM1 hydrochloride
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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
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)
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 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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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 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).
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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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Solvant et solubilité
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)
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.
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.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
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.
Working solution concentration: 0.22 mg/mL
This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
Pureté et documentation
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Fiche technique (274 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Portuguese - PT (252 KB)
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Instruction de manipulation (2659 KB)
Références
[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, 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.
| 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.
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