2407849-89-0
2407849-89-0
Chemical Structure
Votoplam
Synonym(s): PTC518
- CAS No.: 2407849-89-0
- Formula:C21H25N9O
- Molecular Weight:419.48
InChIKey: ICZNVPJUHBURBG-UHFFFAOYSA-N
SMILES: OC1=C(C2=NN=C3C(N=NN3C4CC(C)(C)NC(C)(C4)C)=C2)C=CC(N5N=CC=N5)=C1
Biological Activity: Votoplam (PTC518) is a blood-brain barrier-permeable HTT pre-mRNA inhibitor with an IC50 ≤ 0.1 μM. Votoplam modulates the splicing process of HTT pre-mRNA to include a pseudo-exon carrying a premature translation termination codon, thereby triggering HTT mRNA degradation. Votoplam is applicable to research related to Huntington's disease[1][2][3].
| Cat. No. | Product Name | Purity | Description | Pricing | |||||||||||||||||||
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Votoplam | 99.51% | Votoplam (PTC518) is a blood-brain barrier-permeable HTT pre-mRNA inhibitor with an IC50 ≤ 0.1 μM. Votoplam modulates the splicing process of HTT pre-mRNA to include a pseudo-exon carrying a premature translation termination codon, thereby triggering HTT mRNA degradation. Votoplam is applicable to research related to Huntington's disease. | ||||||||||||||||||||
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References
- [1]. Lee L, et al. Effects of High- and Low-Fat Meals on the Bioavailability and Pharmacokinetics of Votoplam, a HTT Gene Splicing Modifier. Clinical pharmacology in drug development. 2026 Mar;15(3):e1626. [Content Brief]
- [2]. Gao L, et al. Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first-in-human study. British journal of clinical pharmacology. 2024 Dec;90(12):3242-3251. [Content Brief]
- [3]. Sydorenko, et al. Heterocyclic and heteroaryl compounds for treating huntington's disease World Intellectual Property Organization, WO2020005873 A1. 2020-01-02.
