TOKYO UNIVERSITY OF PHARMACY AND LIFE SCIENCES

EpiFrontier Therapeutics Secures $32M AMED Grant to Advance First-in-Class G9a Inhibitor for Beta Globin Disorders

- EpiFrontier Therapeutics received up to $32 million in non-dilutive funding from Japan's AMED to advance EPF-001, a novel G9a inhibitor targeting sickle cell disease and beta thalassemia. - EPF-001 emerged from over a decade of research at RIKEN and Tokyo University, involving screening of 140,000 compounds to identify a highly specific G9a inhibitor that increases fetal hemoglobin expression. - The funding will support Phase 2 clinical development with trials planned primarily at overseas institutions, while patient enrollment in Japan will focus on severe beta thalassemia cases. - The compound has demonstrated significant advantages over standard treatments in preclinical studies in terms of both efficacy and safety, positioning it as a potentially transformative therapy.