Huntingtin Modulator

Votoplam (PTC518) (Example 37) is an HTT gene regulator with an IC₅₀ ≤ 0.1 μM. Votoplam can be used in the research of Huntington's disease.

HTT-D3 is an orally active, blood-brain barrier penetrant splicing modulator of huntingtin (HTT). HTT-D3 promotes the inclusion of a pseudo-exon containing a premature termination codon into HTT pre-mRNA, triggers nonsense-mediated mRNA degradation and reduces HTT protein levels. HTT-D3 induces dose-dependent, comparable reductions in mutant HTT protein in both the brain and peripheral tissues of transgenic mouse models. HTT-D3 can be used for the research of Huntington's disease.

ATXN1-MED15 PPI-IN-1 (compound 5755483) is an inhibitor of ATXN1/MED15. ATXN1-MED15 PPI-IN-1 binds to ATXN1 residues 99-163 and inhibits both the interaction between ATXN1 and MED15, as well as the dimerization of polyQ-expanded ATXN1. ATXN1-MED15 PPI-IN-1 can be used for study of Spinocerebellar ataxia type 1.

Votoplam (PTC518) hydrochloride is an HTT gene regulator with an IC₅₀ ≤ 0.1 μM. Votoplam hydrochloride can be used in the research of Huntington's disease.