| Identification | Back Directory | [Name]
Thieno[3,2-b]pyridine-2-ethanamine, 5-chloro-α,3-dimethyl-7-[(2-thienylmethyl)amino]-, (αS)- | [CAS]
2476724-74-8 | [Synonyms]
PTC258
Thieno[3,2-b]pyridine-2-ethanamine, 5-chloro-α,3-dimethyl-7-[(2-thienylmethyl)amino]-, (αS)- | [Molecular Formula]
C16H18ClN3S2 | [MOL File]
2476724-74-8.mol | [Molecular Weight]
351.92 |
| Chemical Properties | Back Directory | [Boiling point ]
528.6±45.0 °C(Predicted) | [density ]
1.366±0.06 g/cm3(Predicted) | [form ]
Solid | [pka]
8.98±0.10(Predicted) | [color ]
White to yellow |
| Hazard Information | Back Directory | [Uses]
PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model[1]. | [in vivo]
PTC258 (3-24 mg/kg; p.o.; once daily for 3 months) is well tolerated. It corrects the splicing of the ELP1 transcript and significantly increases the amount of functional protein in vivo in all tissues tested, including the brain in mouse model[1]. | Animal Model: | Familial Dysautonomia (FD) Mouse model[1] | | Dosage: | 3 mg/kg, 6 mg/kg, 12 mg/kg, 24 mg/kg | | Administration: | Oral gavage; once daily for 3 month | | Result: | Increased full-length ELP1 transcript in a dose-dependent manner. And also, it increased in functional ELP1 protein in the brain, trigeminal, liver, and quadricep. |
| [References]
[1] Morini E, et al. Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia[J]. bioRxiv, 2022: 2022.11. 04.515198. |
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