SOL784

SOL784 is a first-in-class small molecule inhibitor of **Ubiquitin-Specific Peptidase 14 (USP14)**, currently in Phase 1 development for the treatment of **Fragile X syndrome (FXS)**. Developed by **Sentinel Oncology** in collaboration with the **McQuade Center for Strategic Research and Development (MSRD)**, a subsidiary of **Otsuka**, SOL784 targets the underlying molecular pathology of FXS. The condition is caused by the loss of Fragile X Mental Retardation Protein (FMRP), leading to the dysregulation of protein synthesis and degradation at synapses. By inhibiting USP14, a proteasome-associated deubiquitinating enzyme, SOL784 promotes the degradation of specific proteins that accumulate in the absence of FMRP, potentially restoring synaptic function and improving cognitive and behavioral symptoms. The clinical program includes Phase 1a and Phase 1b trials to evaluate safety, tolerability, and pharmacokinetics in healthy volunteers and patients.