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PT-301

Development stage: Preclinical
Lead developer: Path Therapeutics
Modality: Small Molecules

Overview

PT-301 is a preclinical **small-molecule allosteric modulator of phosphodiesterase 4** being developed by Path Therapeutics for **Dravet syndrome**, a severe epileptic encephalopathy. The program emerged from the company's MitoREAD phenotypic screening platform, which identified PDE4 as a druggable target in CNS disease biology. According to the provided company and program context, PT-301 was designed to address tolerability limitations seen with earlier PDE4-directed approaches and has shown strong preclinical activity, including near-complete seizure freedom in mouse video-EEG studies and restoration of bioenergetic defects to wildtype levels in Dravet mutant zebrafish.

Other names: PT-300 seriesPT300 seriesPT 300 series

Targets

PDE4 (Phosphodiesterase 4A1)

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