Lexeo Therapeutics Reports Promising Early Data for Gene Therapy LX2006 in Friedreich Ataxia Cardiomyopathy

1. Positive Interim Results: Lexeo Therapeutics has announced promising early data from its gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy, a rare and progressive disorder.
2. Clinical Improvements: The interim data showed significant improvements in cardiac biomarkers, including left ventricular mass index (LVMI), left ventricular lateral wall thickness, and high-sensitivity troponin I, indicating a reduction in cardiac hypertrophy and injury.
3. Frataxin Protein Expression: Treatment with LX2006 resulted in increased frataxin protein expression in all evaluated patients, which is crucial for restoring mitochondrial function in myocardial cells.
4. Safety Profile: The gene therapy was well-tolerated with no treatment-related serious adverse events, and all side effects were transient.
5. Future Development: Lexeo plans to explore expedited clinical development, including potential accelerated approval, and will share additional details at an upcoming medical congress.
6. Background: Friedreich ataxia is a genetic disorder caused by mutations in the frataxin gene, affecting approximately 1 in 50,000 people, with 80% of patients eventually developing cardiomyopathy.