RG7800 hydrochloride (Synonyms: RO6885247 hydrochloride) |
| Catalog No.GC37521 |
El clorhidrato de RG7800 es un modulador de empalme SMN2 activo por vÍa oral, con EC1.5xs de 23 nM y 87 nM para el empalme SMN2 y la proteÍna SMN; El clorhidrato de RG7800 tiene el potencial de tratar la atrofia muscular espinal.
Products are for research use only. Not for human use. We do not sell to patients.
Sample solution is provided at 25 µL, 10mM.
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy. EC1.5x: 23 nM (SMN2 splicing), 87 nM (SMN protein)[1]
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; and is the first small molecule SMN2 splicing modifier to enter human clinical trials[1].
RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of δ7 mice[1].
[1]. Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59(13):6086-100.
Animal experiment: | Mice[1]Homozygous Δ7 mice are dosed ip with RG7800 or vehicle (100% DMSO, 2.5 mL/kg) once per day from PND3 through postnatal day 3 (PND23), and the dosing regimen is switched on PND24 to a 3-fold higher oral dose once daily in 0.5% HPMC and 0.1% Tween 80. Litters are randomized across groups. Body weight and survival are assessed daily. Survival analysis is done using GraphPad Prism (log-rank test), and a p <0.05 is considered as significant[1]. |
References: [1]. Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59(13):6086-100. | |
| Cas No. | SDF | ||
| Sinónimos | RO6885247 hydrochloride | ||
| Canonical SMILES | CC1=CC(C2CCN(C)CC2)=CN3C1=NC(C4=NN(C=C(C)N=C5CC)C5=C4)=CC3=O.[xHCl] | ||
| Formula | C24H28N6O.xHCl | M.Wt | |
| Solubility | Water: ≥ 113.5 mg/mL | Storage | Store at -20°C |
| General tips | Please select the appropriate solvent to prepare the stock solution according to the
solubility of the product in different solvents; once the solution is prepared, please store it in
separate packages to avoid product failure caused by repeated freezing and thawing.Storage method
and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored
at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time. |
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| Shipping Condition | Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request. | ||
Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)
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Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )
Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.
Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.
Note: 1. Please make sure the liquid is clear before adding the next solvent.
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3. All of the above co-solvents are available for purchase on the GlpBio website.
Quality Control & SDS
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- Purity: >99.50%
- COA (Certificate Of Analysis)
- SDS (Safety Data Sheet)
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Average Rating: 5 (Based on Reviews and 2 reference(s) in Google Scholar.)
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