Acoramidis Hydrochloride

Acoramidis HydrochlorideOrphan diseases
CAS: 2242751-53-5
MF: C15H18ClFN2O3
MW: 328.77
适应症
Transthyretin amyloidosis; Amyloid Neuropathies, Familial; Cardiomyopathies; Amyloidosis; Polyneuropathies; Amyloidosis, Familial; Heart Diseases; Amyloid Neuropathies
作用靶点
TTR
用途

A transthyretin (TTR) stabilizer. By stabilizing transthyretin (TTR) and inhibiting its misfolding and amyloid deposition, it is used to treat transthyretin amyloid cardiomyopathy (ATTR-CM).

规格标准
>99%
描述

概况

Acoramidis hydrochloride is an effective transthyretin (TTR) stabilizer, a small - molecule agent that acts by stabilizing transthyretin and inhibiting its misfolding as well as amyloid deposition, thus exerting therapeutic effects on transthyretin amyloidosis - related diseases. Acoramidis Hydrochloride can be used in the research of transthyretin amyloid cardiomyopathy (ATTR - CM) and other relevant conditions to explore disease progression mechanisms and therapeutic strategies.

Synonyms: BBP-265; AG-10; ALXN-2060; ALXN 2060; Attruby; Beyonttra; Eidos Therapeutics AG10; AG10 hydrochloride; 3-(3-(3,5-dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid HCl; Acoramidis hydrochloride; AG10 hydrochloride; 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid hydrochloride; Acoramidis?hydrochloride?(AG10 hydrochloride); Acoramidis hydrochloride, 10 mM in DMSO

Product Categories: Acids, Carbocyclic; BCRP/ABCG2 Substrates; Cardiac Therapy; Cytochrome P-450 CYP2C9 Inhibitors; Transthyretin Stabilizers

Mol File: 2242751-53-5.mol

物化性质

Solubility: DMSO: Sparingly Soluble: 1-10 mg/mL

Form: Solid

Color: White to Light Yellow

MSDS信息

实验数据

1. Cell Experiment

Solubility in DMSO : 62.5 mg/mL (190.10 mM; ultrasonic co-solubilization; Hygroscopic DMSO has a significant effect on the solubility of the product, use freshly opened DMSO);H2Solubility in O : < 0.1 mg/mL (insoluble).

Preparing Stock Solutions:

image

Please choose the appropriate solvent to prepare the stock solution according to the solubility of the product in different solvents; Once prepared into a solution, please store it in separate packages to avoid product failure caused by repeated freezing and thawing.Storage method and duration of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)。 Use within 6 months when stored at -80°C, and within 1 month when stored at -20°C.


2. Animal Control

Choose the appropriate dissolution protocol based on your experimental animal and mode of administration.For the following dissolution schemes, please prepare the clarified stock solution according to the in vitro method, and then add the co-solvent in turn:


- In order to ensure the reliability of the experimental results, the clarified stock solution can be properly stored according to the storage conditions; It is recommended that you use the working solution for in vivo experiments and use it on the same day;The percentages shown before the following solvents are the proportion of the volume of that solvent in your final solution; If precipitation and precipitation occur during the preparation process, it can be dissolved by heating and/or ultrasound.


Option 1

Add each solvent in order:10% DMSO,40% PEG300, 5% Tween-80, 45% Saline

Solubility: ≥ 2.08 mg/mL (6.33 mM); Clear solution

A clarified solution of ≥ 2.08 mg/mL (saturation unknown) is obtained for this protocol.

For example, take 1 mL of working solution and add 100 μL of 20.8 mg/mL of clarified DMSO stock solution to 400 μL of PEG300 and mix well. Add 50 μL of Tween-80 to the above system and mix well; Then proceed with 450 μL of normal saline to 1 mL.

Preparation of normal saline: 0.9 g of sodium chloride dissolved in ddH₂O and fixed to 100 mL can obtain a clear and transparent normal saline solution.


Option 2

add each solvent in order: 10% DMSO,90% (20% SBE-β-CD in Saline)

Solubility:≥ 2.08 mg/mL (6.33 mM);Clear solution

A clarified solution of ≥ 2.08 mg/mL (saturation unknown) is obtained for this protocol.

For example, 100 μL of 20.8 mg/mL of clarified DMSO stock solution is added to 900 μL of 20% SBE-β-CD saline in water and mixed well.

2 g of SBE-β-CD (sulfonbutyl ether β-cyclodextrin) powder is set in 10 mL of normal saline and completely dissolved until clear.


Option 3

add each solvent in order: 10% DMSO,90% Corn Oil

Solubility:≥ 2.08 mg/mL (6.33 mM);Clear solution

A clarified solution of ≥ 2.08 mg/mL (saturation unknown) is available for use in animal experiments with an experimental period of more than half a month, as appropriate.

For example, 100 μL of 20.8 mg/mL of clarified DMSO stock solution was added to 900 μL of corn oil and mixed well.

Pharmacodynamics

Mechanism Of Action