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DDC-02
- Development stage
- Preclinical
- Lead developer
- JW Pharmaceutical
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
DDC-02 is an orally administered small-molecule therapeutic candidate discovered by JW Pharmaceutical using its proprietary AI-based drug discovery platform, JWave. It is designed to treat rare genetic neurodevelopmental disorders, with Pitt-Hopkins syndrome (PTHS) as its lead indication, and potential expansion into Fragile X syndrome (FXS) and Rett syndrome (RTT). DDC-02 acts by modulating intracellular signaling pathways involved in neurodevelopment and neural circuit function, specifically by regulating beta-catenin-mediated transcription to normalize deficient Wnt signaling. In preclinical studies, DDC-02 has demonstrated the ability to restore impaired cognitive and behavioral functions to normal levels in adult animal models of these disorders.
02
Targets
CTNNB1 (Beta-catenin)
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