Drug intelligence / Profile preview

LX2021

Development stage
Preclinical
Lead developer
Lexeo Therapeutics
Modality
Gene Therapies
Administration
Intravenous
01

Overview

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].

Other names
Cx43 gene therapyCx-43 gene therapyCx 43 gene therapyAAV-mediated connexin-43 gene therapy
02

Targets

GJA1 (Connexin43 Hemichannel)

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