PTC258 is a specific, orally active modulator of the splicing of the Elongator complex protein 1 gene (ELP1), enhancing its expression both in vitro and in vivo. Additionally, PTC258 has demonstrated good tolerability in a mouse model [1].

PTC258 (administered orally at doses of 3-24 mg/kg once daily for three months) demonstrates good tolerability. It effectively amends the splicing of the ELP1 transcript and substantially enhances the levels of the resultant functional protein across various tissues, brain included, in a mouse model [1].