RNA splicing modulator-4

Cat. No.: HY-177360 Purity: 99.08%: Data Sheet
COA

SDS
Handling Instructions
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RNA splicing modulator-4 is an RNA splicing modulator. RNA splicing modulator-4 controls the inclusion or exclusion of specific exons in precursor mRNA (pre-mRNA) by regulating alternative splicing events, thereby altering the coding sequence and function of mature mRNA. RNA splicing modulator-4 shows promise for research into neurodegenerative diseases (such as Huntington's disease) and cancers.

For research use only. We do not sell to patients.

RNA splicing modulator-4 Chemical Structure

CAS No. : 3067915-73-2

Size		Stock
1 mg		Check price and availability
5 mg		Check price and availability

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Description

In Vitro

RNA splicing modulator-4 (Compound A) binds to the 5' splice site sequence (AGAguaaggg, SEQ ID NO: 1) of HTT pre-mRNA, thereby promoting the inclusion of cryptic toxic exons and inducing transcript degradation via the nonsense-mediated decay pathway^[1].
RNA splicing modulator-4 regulates pre-mRNA splicing at splice site sequences, induces post-transcriptionally unstable gene transcripts, and suppresses gene transcript levels.

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Molecular Weight

397.45

Formula

C₂₁H₂₄FN₅O₂

CAS No.

3067915-73-2

Appearance

Solid

Color

Light yellow to yellow

SMILES

OC1=C(C2=CC=C(N=N2)N([C@@H]3[C@@H]([C@]4([H])N[C@](CCC4)([H])C3)F)C)C=C5C(N=C(O5)C)=C1

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

Powder	-20°C	3 years
	4°C	2 years
In solvent	-80°C	6 months
	-20°C	1 month

Solvent & Solubility

In Vitro:

DMSO : 50 mg/mL (125.80 mM; Need ultrasonic; 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	2.5160 mL	12.5802 mL	25.1604 mL
5 mM	0.5032 mL	2.5160 mL	5.0321 mL
10 mM	0.2516 mL	1.2580 mL	2.5160 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, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

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

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

This equation is commonly abbreviated as: C₁V₁ = C₂V₂

Concentration _(start)

Volume _(start)

Concentration _(final)

Volume _(final)

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: ≥ 2.5 mg/mL (6.29 mM); Clear solution

This protocol yields a clear solution of ≥ 2.5 mg/mL (saturation unknown).
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (25.0 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% Corn Oil
Solubility: ≥ 2.5 mg/mL (6.29 mM); Clear solution

This protocol yields a clear solution of ≥ 2.5 mg/mL (saturation unknown). If the continuous dosing period exceeds half a month, please choose this protocol carefully.
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (25.0 mg/mL) to 900 μL Corn oil, and mix evenly.

In Vivo Dissolution Calculator

Please enter the basic information of animal experiments:

Dosage

mg/kg

Animal weight
(per animal)

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).

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: 99.08%

Select Batch:

Data Sheet (272 KB) SDS (252 KB)

COA (249 KB) HNMR (268 KB) LCMS (325 KB)

Handling Instructions (2659 KB)

References

[1]. Wester DE POEL, et al. Crystalline forms of 6-(6-(((1r,2r,3s,5s)-2-fluoro-9-azabicyclo[3.3.1]nonan-3-yl)(methyl)amino)pyridazin-3-yl)-2-methylbenzo[d]oxazol-5-ol, a splicing modulator for the treatment of huntington's disease. WO2025006887A1. 2025-01-02.

Complete Stock Solution Preparation Table

Optional Solvent	Concentration Solvent Mass	1 mg	5 mg	10 mg	25 mg

DMSO	1 mM	2.5160 mL	12.5802 mL	25.1604 mL	62.9010 mL
	5 mM	0.5032 mL	2.5160 mL	5.0321 mL	12.5802 mL
	10 mM	0.2516 mL	1.2580 mL	2.5160 mL	6.2901 mL
	15 mM	0.1677 mL	0.8387 mL	1.6774 mL	4.1934 mL
	20 mM	0.1258 mL	0.6290 mL	1.2580 mL	3.1450 mL
	25 mM	0.1006 mL	0.5032 mL	1.0064 mL	2.5160 mL
	30 mM	0.0839 mL	0.4193 mL	0.8387 mL	2.0967 mL
	40 mM	0.0629 mL	0.3145 mL	0.6290 mL	1.5725 mL
	50 mM	0.0503 mL	0.2516 mL	0.5032 mL	1.2580 mL
	60 mM	0.0419 mL	0.2097 mL	0.4193 mL	1.0483 mL
	80 mM	0.0315 mL	0.1573 mL	0.3145 mL	0.7863 mL
	100 mM	0.0252 mL	0.1258 mL	0.2516 mL	0.6290 mL