Epizyme has assembled a world-class team with a passion for scientific innovation and a commitment to developing novel epigenetic therapies for patients living with cancer and other serious diseases. Epizyme is pioneering the discovery and development of small molecule inhibitors of histone methyltransferases (HMTs), histone acetyltransferases (HATs) and helicases.

Epigenetics refers to a broad biological regulatory system that controls gene expression without altering the makeup of the genes themselves. Gene expression is regulated by HMTs and other chromatin modifying proteins (CMPs). Abnormal expression of genes that either promote cell growth and survival or that act as tumor suppressors can be caused by errors in CMP function. These genetic changes to CMP function can instigate different types of cancers and other diseases. By controlling gene expression, epigenetics has the potential to play an important role in treating cancer and other serious diseases. To that end, while we have always been focused on oncology, recent compelling research from the academic community strongly implicates G9a, an HMT, as a potential drug target for sickle cell disease (SCD).

Our scientific approach includes using CRISPR, a gene editing tool, to efficiently identify targets where we see a clear signal for selective activity in specific tumor types. This approach, in combination with our comprehensive library of tumor cell lines, has led us to a compelling set of HMTs, HATs and helicases, targets of high interest in cancer. To prioritize the targets and programs we want to move forward, we use a comprehensive, multi-factor assessment approach. We then utilize an integrated combination of biochemistry, biology, structural biology, DMPK, safety sciences and medicinal chemistry to optimize compound development and generate novel product candidates that may be advanced for the treatment of multiple types of cancer and other serious diseases.