LEXEO Therapeutics Announces FDA Clearance of Investigational New Drug Application for LX2006, an AAV-based Gene Therapy Candidate for Friedreich’s Ataxia Cardiomyopathy

NEW YORK, Feb. 16, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics.

LEXEO today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LX2006. LX2006 is an AAV-based gene therapy candidate designed to intravenously deliver a functional frataxin gene, for the treatment of Friedreich’s ataxia cardiomyopathy (FA cardiomyopathy). LEXEO plans to initiate this open-label, dose-escalation Phase 1/2 clinical trial of LX2006 in patients with FA cardiomyopathy in the middle of 2022. 

The Phase 1/2 study is a 52-week, dose-ascending, open-label trial of LX2006 in patients who have FA cardiomyopathy. LX2006 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts of five patients each.

FDA Maintains Clinical Hold on Larimar’s Friedreich’s Ataxia Study

Global Genes. February 15, 2022. Larimar Therapeutics, a company focused on developing treatments for complex rare diseases, said that the U.S. Food and Drug Administration is maintaining the clinical hold on Larimar’s CTI-1601 program for the treatment of Friedreich’s ataxia.