GB-601

GB-601 is a novel, long-acting cyclic guanosine monophosphate (cGMP) analog currently in preclinical development for the treatment of inherited retinal diseases, including retinitis pigmentosa, Leber's congenital amaurosis, and Stargardt’s disease. Unlike existing therapies that target specific genetic mutations, GB-601 is designed to be **mutation-agnostic**, aiming to address a broad range of over 280 genetic mutations responsible for such diseases. The drug works by leveraging cGMP signaling pathways, which are implicated in retinal degeneration, to provide therapeutic benefit. GB-601’s unique formulation offers extended ocular exposure, potentially reducing the need for frequent dosing. It is being developed by Graybug Vision, based on technologies acquired from Mireca Medicines GmbH[3][5][7].