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LX2021 is a gene therapy candidate developed by Lexeo Therapeutics for the treatment of inherited cardiac muscle disorders associated with a high risk of sudden death, including arrhythmogenic cardiomyopathy (ACM) and certain forms of dilated cardiomyopathy. The therapy is designed to deliver a functional connexin 43 (Cx43) protein, an integral component of gap junctions in cardiac muscle cells. Cx43 enables electrical synchronization necessary for proper heart contraction. In ACM and related conditions, loss or mislocalization of Cx43 at cell junctions leads to impaired electrical conduction and increased risk of arrhythmias and sudden death. By restoring Cx43 expression at cardiac cell-cell junctions, LX2021 aims to address the underlying molecular deficit common across multiple genetic causes of ACM, including desmoplakin (DSP) cardiomyopathy[1][3][5].
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