Drug intelligence / Profile preview

Cx43-S3E

Development stage
Preclinical
Lead developer
Rutgers University
Modality
Gene Therapies, Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

Cx43-S3E is a phospho-mimetic variant of Connexin 43 (Cx43), a gap junction protein responsible for electrical signal propagation in the heart. In Duchenne muscular dystrophy (DMD), Cx43 is typically upregulated and undergoes pathological remodeling, moving away from the intercalated discs, which contributes to cardiac fibrosis and arrhythmias. Cx43-S3E was engineered by replacing a specific triplet of serine residues with glutamic acid to mimic a constitutively phosphorylated state. Preclinical studies in *mdx* mouse models have demonstrated that Cx43-S3E prevents Cx43 remodeling and the subsequent development of fibrotic cardiomyopathy, arrhythmias, and heart failure. It is being investigated as a potential therapeutic target or gene therapy candidate for the treatment of DMD-associated cardiomyopathy.

Other names
phospho-mimic Connexin 43Cx43-S3E mutantCx-43-S3E mutantCx 43-S3E mutant
02

Targets

GJA1 (Connexin43 Hemichannel)

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