| Field | Specification |
|---|---|
| Alternative names | PTC518 |
| CAS no. | |
| Applications | |
| Molecular weight | |
| Molecular formula | C21H25N9O |
| Purity | |
| SMILES | |
| Form | Solid |
| Storage | |
| Shipping | |
| Catalog no. (Mfr.) | |
| Main SKU |
Compound Overview
Votoplam (PTC518) is a blood-brain-barrier-permeable inhibitor of HTT pre-mRNA, with an IC50 ≤0.1 μM. It modulates HTT pre-mRNA splicing to include a pseudo-exon carrying a premature translation termination codon, triggering HTT mRNA degradation, and it is applicable to Huntington's disease research[1][2][3]. It is supplied as a light yellow to yellow solid (C21H25N9O, MW 419.48) at 99.51% purity.
Physical & Chemical Properties
| CAS Number | 2407849-89-0 |
|---|---|
| Molecular Formula | C21H25N9O |
| Molecular Weight | 419.48 g/mol |
| Purity | 99.51% |
| Appearance | Solid |
| Color | Light yellow to yellow |
| SMILES | OC1=C(C2=NN=C3C(N=NN3C4CC(C)(C)NC(C)(C4)C)=C2)C=CC(N5N=CC=N5)=C1 |
| Signaling Pathway | Neuronal Signaling |
| Solubility | In Vitro: DMSO: 12.5 mg/mL (29.80 mM; Requires sonication and warming and heat to 60°C; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO) |
| Storage | Powder: -20°C, 3 years; 4°C, 2 years. In solvent: -80°C, 6 months; -20°C, 1 month. |
| Shipping | Room temperature in continental US; may vary elsewhere. |
Literature Cited
Sources cited in this description and in the In Vitro & In Vivo Data tab. Peer-reviewed publications that used this product are listed under References.
[3]. Sydorenko, et al. Heterocyclic and heteroaryl compounds for treating huntington's disease World Intellectual Property Organization, WO2020005873 A1. 2020-01-02.
Safety
For Research Use Only. Not for use in diagnostic or therapeutic procedures, and not for human or veterinary use. Handle in accordance with the Safety Data Sheet and your institution's chemical hygiene plan.
In Vitro
| Solvent | Solubility | Notes |
|---|---|---|
| DMSO | 12.5 mg/mL (29.80 mM) | requires sonication and warming and heat to 60°C; use freshly opened DMSO (absorbed moisture lowers solubility) |
Aliquot the stock solution and store it at -80°C (up to 6 months) or -20°C (up to 1 month); avoid repeated freeze-thaw cycles.
In Vivo
Choose the formulation that suits the animal model and route of administration; percentages are volume ratios of the final working solution. Start from a clear DMSO stock (see In Vitro above), add the co-solvents one at a time in the order listed, mixing after each addition, and prepare the working solution fresh on the day of dosing. If precipitation or phase separation occurs, gentle warming or sonication can help.
Protocol 1
| Composition | 10% DMSO + 40% PEG300 + 5% Tween-80 + 45% saline |
|---|---|
| Result | ≥ 1.25 mg/mL (2.98 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 400 μL PEG300; then 50 μL Tween-80; then 450 μL saline to bring the volume to 1 mL. Saline: dissolve 0.9 g sodium chloride in ddH2O and make up to 100 mL. |
Protocol 2
| Composition | 10% DMSO + 90% (20% SBE-β-CD in saline) |
|---|---|
| Result | ≥ 1.25 mg/mL (2.98 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 900 μL 20% SBE-β-CD in saline. 20% SBE-β-CD in saline: dissolve 2 g SBE-β-CD powder in 10 mL saline until clear (4°C, store up to one week). |
Protocol 3
| Composition | 10% DMSO + 90% Corn Oil |
|---|---|
| Result | ≥ 1.25 mg/mL (2.98 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). Use with caution if continuous dosing will exceed two weeks. For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 900 μL corn oil. |
Data provided by the manufacturer.
In Vitro
In liver microsomes and hepatocytes, the metabolic profiles of Votoplam are unremarkable, and major CYP enzymes or transporters are not inhibited by it[2]. Plasma protein binding of Votoplam ranges from 80% to 88%[2]. As an HTT gene modulator, Votoplam has an IC50 ≤ 0.1 μM[3].
In Vivo
In transgenic mouse models of Huntington's disease, Votoplam downregulates HTT protein in multiple tissues in a dose-dependent manner[2].
Data provided by the manufacturer. Numbered citations refer to the Literature Cited list in the product description.
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