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

Branaplam  (Synonyms: LMI070; NVS-SM1)

製品番号: HY-19620 純度: 98.90%
COA 取扱説明書 Technical Support

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 番号 : 1562338-42-4

容量 価格（税別） 在庫状況 数量
>無料サンプル (0.1 - 0.2 mg)   今すぐ申し込む  
5 mg $120 在庫あり
10 mg $190 在庫あり
25 mg $326 在庫あり
50 mg $490 在庫あり
100 mg $690 在庫あり
200 mg $966 在庫あり
500 mg $1600 在庫あり
1 g   お問い合わせ  
5 g   お問い合わせ  

* アイテムを追加する前、数量をご選択ください

This product is a controlled substance and not for sale in your territory.

カスタマーレビュー

Based on 15 publication(s) in Google Scholar

Other Forms of Branaplam:

Branaplam 関連抗体

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.

    DNA/RNA Synthesis アイソフォーム固有の製品をすべて表示:

    すべてのアイソフォームを表示
    RNASE L DNA Polymerases RNA Polymerases Helicases DNA Ligase

    • 生物活性

    • 純度とドキュメンテーション

    • 参考文献

    • カスタマーレビュー

    製品説明

    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]
    体外実験

    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 関連抗体
    体内実験

    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.
    分子量

    393.48

    分子式

    C22H27N5O2
    CAS 番号
    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
    輸送条件

    Room temperature in continental US; may vary elsewhere.
    保管条件

    4°C, sealed storage, away from moisture

    *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
    溶剤 & 溶解度
    体外: 

    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
    10 mM --- --- ---
    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
    =
    Concentration
    ×
    Volume
    ×
    Molecular Weight *

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

    一般には略語で表示されます：C1V1 = C2V2

    濃度 （開始）

    C1

    ×
    体積 （開始）

    V1

    =
    濃度 （終了）

    C2

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    体積 （終了）

    V2

    体内:

    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.
    純度とドキュメンテーション

    純度: 98.90%

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

    取扱説明書 (2659 KB)
    参考文献

    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

    • Molarity Calculator

    • Dilution Calculator

    The molarity calculator equation

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

    質量   濃度   体積   分子量 *
    = × ×

    The dilution calculator equation

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

    一般には略語で表示されます：C1V1 = C2V2

    濃度 （開始） × 体積 （開始） = 濃度 （終了） × 体積 （終了）
    × = ×
    C1   V1   C2   V2
    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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    製品名:
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    製品番号:
    HY-19620
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