Mid-Year Clinical Update Expected to Include:
Safety and tolerability data for all participants dosed across both the SUNRISE-FA and Weill Cornell clinical trials (at least 16 participants, including 6 participants with abnormal LVMI at baseline)
Pre- and post-treatment cardiac frataxin protein expression measured via LCMS for all four participants at the highest dose (1.2x1012 vg/kg, Cohort 3)
Clinical biomarker data, including left ventricular mass index (LVMI), left ventricular wall thickness and high-sensitivity troponin I, for participants with >6-months of follow up
Functional and patient-reported outcome data for participants with >6-months of follow up
Safety: LX2006 continues to be generally well tolerated with no new treatment-related serious adverse events to report