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 at a concentration of 50 μM disrupts G-rich RNA and EXOSC3 interactions by approximately 18% and triggers notable spinal curvature, mirroring the PCH1B phenotype in zebrafish embryos. Additionally, this compound significantly increases ataxin1b mRNA levels (~6-fold) while moderately increasing ataxin1a mRNA levels in embryos. Furthermore, ERD03 leads to a reduction in zebrafish cerebellum size, resulting in cerebella that are half the size of those in control groups treated with DMSO.