Clinical-stage genetic medicines company leveraging gene therapy to address devastating cardiac diseases with no existing disease-modifying treatments.

Our lead-clinical stage program LX2006 (frataxin gene replacement therapy) targets the cardiomyopathy associated with Friedreich's Ataxia, the leading cause of death for ~5,000 US patients. 

Please note that we are not accepting requests from CRO, CDMO and other service organizations at this time.