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Chemical Structure : Votoplam hydrochloride
Catalog No.: PC-23056Not For Human Use, Lab Use Only.
Votoplam (PTC518) hydrochloride is a small molecule HTT gene modulator and splicing modifier, selectively lowers huntingtin mRNA and protein.
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Votoplam (PTC518) hydrochloride is a small molecule HTT gene modulator and splicing modifier, selectively lowers huntingtin mRNA and protein.
Votoplam (PTC518) promotes the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels.
Votoplam (PTC518) demonstrated a dose-dependent lowering of HTT mRNA and protein to the targeted 30-50% reduction in a Phase 1 healthy volunteer study of PTC518 for Huntington's disease.
| M.Wt | 455.95 | |
| Formula | C21H26ClN9O | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Patent WO2020005873 A1.
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