This morning, we were pleased to share positive interim clinical data for LX2006, our investigational gene therapy, that includes both cardiac and neurological outcome measures. LX2006 (AAVrh10hFXN) is currently being studied in the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine investigatorinitiated Phase 1A trial for the treatment of Friedreich ataxia cardiomyopathy.
Thursday, October 9, 2025
LEXEO Message to the Friedreich Ataxia Community
Message to the Friedreich Ataxia Community. October 7, 2025.
Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons
Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons
Olivier Griso, Deepika Mokkachamy Chellapandi, Amelie Weiss, Ioannis Manolaras, Helene Puccio. bioRxiv 2025.10.07.680891; doi:10.1101/2025.10.07.680891
Our findings uncover AMPK-mTOR dysregulation as a key driver of neuronal growth impairment in FA. This robust neuronal model provides new insights into proprioceptive neuron vulnerability and offers a platform for therapeutic discovery.
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