On March 24th 2025, the pharmaceutical company Lexeo Therapeutics provided a number of updates on their phase 1/2 trial of the gene therapy drug LX2006 for Friedreich’s ataxia (FA) cardiomyopathy.
Cardiomyopathy is a disease of the heart involving stretching, thickening, or stiffening of the heart muscle, making it harder to pump blood around the body.
Frataxin is a protein that is deficient in people with FA. LX2006 is designed to transfer the FXN gene (which codes for the frataxin protein) to heart cells and increase frataxin protein levels in the energy centres of cells called mitochondria. The increase in frataxin levels in the mitochondria restores energy production in heart cells.
Lexeo have announced a number of important updates to their LX2006 programme, which they have shared with the FA community here.
Read more about the results from the phase 1/2 trial of LX2006 in FA cardiomyopathy here.
