LEXEO Therapeutics is a gene therapy company, developing treatments for a number of conditions including cardiomyopathy associated with FA.
In February 2022, LEXEO Therapeutics announced that the FDA had cleared an Investigational New Drug application for their gene therapy product LX2006. This is a requirement before clinical trials can take place.
LEXEO are planning to start a Phase I/II clinical trial later this year in people with FA cardiomyopathy. Once receiving LX2006, patients will be followed for safety and efficacy (initial 12-month observation followed by long-term follow-up for a total of 5 years). This trial will be carried out on 10 patients in the US, and will mainly be evaluating the safety of LX2006. If this trial is successful, LEXEO hopes to conduct future trials in other regions.
LX2006 is a gene therapy treatment, which is designed to deliver a functional frataxin gene to the cells of the heart, to promote expression of the frataxin protein, which is reduced in FA. In preclinical studies, LX2006 reversed the cardiac symptoms of FA in animal models.
LEXEO Therapeutics are planning to hold patient webinars in the future to share updates with the FA patient community. We are pleased to see this exciting development which will see a FA gene therapy candidate enter clinical studies, and we will report any results as they are released.
Read the press release here.