NEW YORK – Lexeo Therapeutics on Tuesday said it has begun treating patients with Friedreich's ataxia cardiomyopathy with the gene therapy candidate LX2006 in a Phase I/II trial.
LX2006 is an investigational adeno-associated virus vector-based gene therapy under development for this inherited neuromuscular disorder caused by deficiency in the frataxin protein. LX2006 delivers a functional FXN gene and is designed to promote expression of frataxin and restore mitochondrial function in myocardial cells.
New York-based Lexeo is evaluating the gene therapy in the multicenter open-label SUNRISE-FA trial, in which patients receive a one-time intravenous infusion in at least two ascending-dose cohorts. Investigators started dosing patients in this second cohort of patients in the study after determining LX2006 was "well tolerated" in an initial cohort of patients who had no unexpected adverse events or toxicities, the company said.
Lexeo expects to report an initial data readout from these first two cohorts, including data on myocardial protein expression and serum and cardiac structure and function biomarkers, in the first half of 2024.