Lexeo Therapeutics, Inc. announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to LX2006, the company?s AAVrh.10hFXN-based gene therapy candidate for the treatment of Friedreich?s ataxia (FA) cardiomyopathy. LX2006 is designed to deliver a functional frataxin gene to promote frataxin protein expression and restore mitochondrial function in myocardial cells. Fast Track is a process designed to facilitate the development and expedite the review of new drugs intended to treat serious conditions and address unmet medical need.

This designation was granted based on available preclinical data. SUNRISE-FA, a Phase 1/2 multicenter, 52-week, dose-ascending, open-label clinical trial, is ongoing to evaluate the safety and tolerability, as well as preliminary efficacy, of LX2006 in patients with FA cardiomyopathy. LX2006 is administered as a one-time intravenous infusion to patients in at least two ascending-dose cohorts with the potential for a third cohort.

Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial year of the trial, resulting in data from a total of five years post-LX2006 treatment.