2. Cell Cycle/DNA Damage Membrane Transporter/Ion Channel
  3. DNA/RNA Synthesis Potassium Channel
  4. Branaplam

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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CAS No. : 1562338-42-4

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Based on 15 publication(s) in Google Scholar

Other Forms of Branaplam:

Branaplam Related Antibodies

Top Publications Citing Use of Products

    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).

    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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    • Biological Activity

    • Purity & Documentation

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    Description

    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[1][2].

    IC50 & Target

    IC50: 20 nM (SMN)[1]
    EC50: 6.3 μM (hERG)[2]
    In Vitro

    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 Related Antibodies
    In Vivo

    Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) 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) 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.

    Animal Model: C/+ SMA mouse model[1]
    Dosage: 3, 10, 30 mg/kg
    Administration: Oral
    Result: Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord.
    Animal Model: Male Sprague-Dawley rat[2]
    Dosage: 1 mg/kg (IV);3 mg/kg (PO) (Pharmacokinetic Analysis)
    Administration: IV or PO
    Result: Had a CL of 25 mL/min/kg and an AUC of 3.03 μM•h.
    Molecular Weight

    393.48

    Formula

    C22H27N5O2
    CAS No.
    Appearance

    Solid

    Color

    White to yellow

    SMILES

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

    Room temperature in continental US; may vary elsewhere.
    Storage

    4°C, sealed storage, away from moisture

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

    Solvent & Solubility
    In Vitro: 

    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)

    Preparing
    Stock Solutions
    Concentration Solvent Mass 1 mg 5 mg 10 mg
    1 mM 2.5414 mL 12.7071 mL 25.4143 mL
    5 mM 0.5083 mL 2.5414 mL 5.0829 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, 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.

    • Molarity Calculator

    • Dilution Calculator

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

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

    This equation is commonly abbreviated as: C1V1 = C2V2

    Concentration (start)

    C1

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    Volume (start)

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    C2

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    In Vivo:

    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.

    • Protocol 1

      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.
    • Protocol 2

      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.

    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.

    • Protocol 1

      Add each solvent one by one:  50% PEG300    50% Saline

      Solubility: 5 mg/mL (12.71 mM); Suspended solution; Need ultrasonic

    In Vivo Dissolution Calculator
    Please enter the basic information of animal experiments:

    Dosage

    mg/kg

    Animal weight
    (per animal)

    g

    Dosing volume
    (per animal)

    μL

    Number of animals

    Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.
    Please enter your animal formula composition:
    %
    DMSO +
    % 
    +
    %
    Tween-80 +
    %
    Saline
    Recommended: Keep the proportion of DMSO in working solution below 2% if your animal is weak.
    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).
    Calculation results:
    Working solution concentration: 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)

    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).
    Method for preparing in vivo working solution for animal experiments: Take μL DMSO stock solution, add μL . μL , mix evenly, next add μL Tween 80, mix evenly, then add μL Saline.
     If the continuous dosing period exceeds half a month, please choose this protocol carefully.
    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.
    Purity & Documentation

    Purity: 98.90%

    SDS (393 KB)

    COA (248 KB) HNMR (121 KB) LCMS (91 KB)

    Handling Instructions (2659 KB)
    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, 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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    Branaplam 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:

    Branaplam1562338-42-4LMI070 NVS-SM1LMI 070LMI-070DNA/RNA SynthesisPotassium ChannelKcsAorallysurvivalmotorneuron-2SMN2splicingSMNhuman-ether-a-go-go-relatedgenehERGspinalmuscularatrophySMAInhibitorinhibitorinhibit

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