EXO-TBKi

EXO-TBKi is a novel, orally bioavailable small molecule that acts as a selective exosite inhibitor of the serine/threonine-protein kinase TBK1. Unlike traditional active-site inhibitors, EXO-TBKi targets an exosite on TBK1, disrupting its recruitment by STING and selectively blocking downstream activation of type I interferon (IFN) and NF-κB responses. This mechanism allows for potent inhibition of pathogenic effects in autoimmune diseases while sparing beneficial antiviral and autophagy functions. Preclinical studies have demonstrated efficacy in cellular models, mouse models (including TREX1 knockout mice), and patient-derived samples from diseases such as systemic lupus erythematosus (SLE), systemic sclerosis (scleroderma), cutaneous lupus erythematosus (CLE), Sjögren’s syndrome, and Aicardi-Goutieres syndrome. The drug is being developed by Exo Therapeutics[1][2][3][5][7].

Other names

TBK1 exosite inhibitorTBK-1 exosite inhibitorTBK 1 exosite inhibitor