Epizyme Presents Preclinical Data on Novel G9a Program and Introduces Next Drug Development Candidate at the American Society of Hematology Annual Meeting
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 sickle cell disease and β-thalassemia. To this end, multiple academic groups have previously discovered that inhibition of the histone methyltransferase (HMT) G9a leads to increased levels of fetal hemoglobin in preclinical in vitro studies. Building upon these findings, scientists from
“Sickle cell disease is considered one of the first genetically defined diseases, yet despite all the advances of modern medicine, it remains an area of significant medical need,” said
Based on its research efforts,
"We are excited about the potential of our novel, internally discovered program,” said
The oral presentation provides details on Epizyme’s tool compound that supports further study of G9a inhibition and the reactivation of fetal hemoglobin (abstract #537): Reawakening of Human Fetal Hemoglobin and an Epigenetic Path to the
About Sickle Cell Disease
Sickle cell disease (SCD) is an inherited red blood cell disorder. People with SCD have abnormal hemoglobin, called hemoglobin S or sickle hemoglobin, in their red blood cells. A monogenic disease, SCD is most common in people with African ancestry. Approximately 300,000 people have sickle cell disease globally, with an estimated 150,000 of those patients located in the U.S. and
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Source: Epizyme, Inc.