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Simufilamum Dihydrochloride Monohydrate

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Catalog No. T202428Cas No. 2375909-85-4
Alias Simufilam 2HCl,H2O, CJ368X8WT6 2HCl,H2O

Simufilamum (also known as PTI-125) is an oral small-molecule compound candidate that targets altered conformations of filamin A. It binds and reverts the misfolded filamin A in the brains of Alzheimer's disease patients, thereby slowing the disease's pathological progression. Additionally, PTI-125 reduces tau protein hyperphosphorylation, decreases Aβ42 aggregation and deposition, neurofibrillary tangles, and neuroinflammation. The compound demonstrates concentration-dependent efficacy in vitro, starting at 1 picomolar (pM), in both control groups and post-mortem hippocampal slices from Alzheimer's disease (AD) patients. PTI-125 is the first therapeutic candidate to preferentially bind and reverse pathogenic protein conformations.

Simufilamum Dihydrochloride Monohydrate

Simufilamum Dihydrochloride Monohydrate

😃Good
Catalog No. T202428Alias Simufilam 2HCl,H2O, CJ368X8WT6 2HCl,H2OCas No. 2375909-85-4
Simufilamum (also known as PTI-125) is an oral small-molecule compound candidate that targets altered conformations of filamin A. It binds and reverts the misfolded filamin A in the brains of Alzheimer's disease patients, thereby slowing the disease's pathological progression. Additionally, PTI-125 reduces tau protein hyperphosphorylation, decreases Aβ42 aggregation and deposition, neurofibrillary tangles, and neuroinflammation. The compound demonstrates concentration-dependent efficacy in vitro, starting at 1 picomolar (pM), in both control groups and post-mortem hippocampal slices from Alzheimer's disease (AD) patients. PTI-125 is the first therapeutic candidate to preferentially bind and reverse pathogenic protein conformations.
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Product Introduction

Bioactivity
Description
Simufilamum (also known as PTI-125) is an oral small-molecule compound candidate that targets altered conformations of filamin A. It binds and reverts the misfolded filamin A in the brains of Alzheimer's disease patients, thereby slowing the disease's pathological progression. Additionally, PTI-125 reduces tau protein hyperphosphorylation, decreases Aβ42 aggregation and deposition, neurofibrillary tangles, and neuroinflammation. The compound demonstrates concentration-dependent efficacy in vitro, starting at 1 picomolar (pM), in both control groups and post-mortem hippocampal slices from Alzheimer's disease (AD) patients. PTI-125 is the first therapeutic candidate to preferentially bind and reverse pathogenic protein conformations.
AliasSimufilam 2HCl,H2O, CJ368X8WT6 2HCl,H2O
Chemical Properties
Molecular Weight350.28
FormulaC15H25Cl2N3O2
Cas No.2375909-85-4
Storage & Solubility Information
StoragePowder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice.

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Mother liquor preparation method: 2 mg of drug dissolved in 50 μL DMSOTargetMol | reagent (mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.
Preparation method for in vivo formula: Take 50 μL DMSOTargetMol | reagent main solution, add 300 μLPEG300TargetMol | reagent mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLddH2OTargetMol | reagent mix well and clarify
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