ERD03, with a dissociation constant (Kd) of 17±7 μM, effectively disrupts the EXOSC3-RNA interaction. It has been shown to induce a PCH1B-like phenotype in zebrafish embryos, making it a valuable tool for research into neurological disorders [1].

ERD03 inhibits the binding of G-rich RNA and EXOSC3 with ~18% inhibition at 50 μM [1].ERD03 (50 μM; 5 days) causes significant spinal curvature and recapitulates PCH1B phenotype in development in zebrafish embryos [1].ERD03 (50 μM) induces a ~6-fold upregulation of ataxin1b mRNA and results in a minor accumulation of ataxin1a mRNA in embryos. ERD03 induces an atrophy of the zebrafish cerebellum and results in zebrafish cerebella half the size of control brains in DMSO [1].