Chrexanthomycin A is a marine natural product with notable biological activity and oral bioavailability. It exhibits binding affinities to DNA (G4C2)4G4 and RNA (G4C2)2G4 with Kd values of 2.2 mM and 3.0 mM, respectively. Chrexanthomycin A can be utilized for research on neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).

Chrexanthomycin A (Compounds cA) effectively binds to DNA (G4C2) 4 G4 and RNA (G4C2) 2 G4, with dissociation constants (K d) of 2.2 mM and 3.0 mM, respectively. It demonstrates high cell permeability and low cytotoxicity. At 1.61 μM, Chrexanthomycin A rescues G4C2 EHR-related cellular pathologies. It selectively interacts with G4C2 G4 structures in both DNA and RNA. In a concentration range of 0-100 μM, it significantly reduces cell death induced by G4C2 EHR, diminishes RNA foci in (G4C2)29-expressing Neuro2a cells, and effectively reduces reactive oxygen species (ROS) in HT22 cells. In a Cell Cytotoxicity Assay on HEK293T cells at concentrations of 0.16, 1.61, and 16.13 μM for 24 hours, it showed no cytotoxicity up to 10 μg/mL.

Chrexanthomycin A (Compounds cA) at a concentration of 100 μM, administered through feeding, effectively mitigates eye degeneration and enhances locomotor performance in subjects expressing (G4C2)29.