Drug intelligence / Profile preview

Exo-CHP

Development stage
Preclinical
Modality
Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

Exo-CHP is an engineered exosome-based therapy derived from mesenchymal stromal cells (MSCs) designed for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. These exosomes are synthesized using click chemistry and are characterized by their miRNA content. Exo-CHP is specifically engineered to target the myocardium, where it mitigates pathological progression by reducing inflammation and fibrosis and improving cardiac function. Its therapeutic effects are mediated through the regulation of gene expression at the transcriptomic level, the prevention of dystrophin protein complex degradation, and the inhibition of intracellular calcium influx and calpain protease activity. Preclinical studies in mouse models have demonstrated its feasibility and efficacy as a cell-free therapeutic intervention.

Other names
engineered mesenchymal stromal cell-derived exosomes
02

Targets

DMD (Dystrophin)CAPN (Schistosoma calpain large subunit family)

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