LYS-FXS01

**LYS-FXS01** is an investigational gene therapy developed by Lysogene for the treatment of **Fragile X Syndrome (FXS)**, a genetic neurodevelopmental disorder caused by mutations in the FMR1 gene leading to loss of fragile X mental retardation protein (FMRP). It targets **DGKK (diacylglycerol kinase kappa)**, a protein implicated in dysregulated signaling pathways downstream of metabotropic glutamate receptor 1 (mGluR1) in FXS pathophysiology. The therapy is delivered via an AAV vector, leveraging Lysogene's CNS-targeted gene therapy platform, with preclinical studies initiated following a 2020 collaboration with Yeda (Weizmann Institute of Science). Lysogene has secured non-dilutive financing, including €4.3M from Bpifrance and part of a €15M EIB loan, to support preclinical advancement, positioning it as a potential disease-modifying treatment addressing core synaptic and behavioral deficits in FXS.[1][7][8][9][11]