Epizyme is developing EZM8266, a G9a inhibitor, for the potential treatment of sickle cell disease (SCD).
It is widely understood within the SCD research community that elevation of fetal hemoglobin, which is normally silenced after birth, has disease-modifying potential for patients with β-globinopathies, such as SCD and β-thalassemia. To this end, multiple academic groups have previously discovered that inhibition of the histone methyltransferase (HMT) G9a led to increased levels of fetal hemoglobin in preclinical in vitro studies.
Building upon these findings, scientists from Epizyme leveraged their expertise in HMT drug discovery to generate potent, selective inhibitors of G9a with drug-like properties.
EZM8266 will begin IND-enabling studies in 2018.