Diseases We Target

Starting with Cancer, We Are Going After Diseases that Have Largely Eluded Other Targeted Approaches

We have one investigational medicine in a Phase 2 clinical trial in genomically defined subsets of patients with acute myeloid leukemia (AML). We are also building a pipeline of programs in earlier stages of research for cancer, as well as genetic diseases, that lack effective treatment options.

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia. Although several new therapies have been introduced to treat AML and a number of investigational therapies are currently in clinical trials, there remains a significant medical need for additional targeted approaches since AML is driven by many different genomic mutations and alterations in different patients. We are focused on subsets of AML patients with highly specialized regulatory regions of DNA, known as super-enhancers, associated with either the RARA or IRF8 gene, or both. By driving abnormally high expression of these genes, the RARA and IRF8 super-enhancers lock cells in an immature and undifferentiated state. These undifferentiated cells, often called blast cells, multiply rapidly and prevent the production of healthy blood cells. We estimate that about one-third of all AML patients have either the RARA or IRF8 super-enhancer, or both.

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