Diseases We Target

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

We have two investigational medicines in clinical trials, one in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) and the other in patients with advanced solid tumors, initially focusing on ovarian cancer. We are also building a pipeline of programs in earlier stages of research for additional cancers, 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. There have been relatively few advances in treatment of AML over the past 20 years, with chemotherapies, stem cell transplantation or hypomethylating agents remaining the standard of care for many patients. Although a number of investigational therapies are currently in clinical trials and several targeted agents or reformulated versions of chemotherapy have been introduced to treat AML, 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.

Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (MDS) is a family of blood disorders in which the bone marrow fails to make enough healthy red blood cells, white blood cells or platelets. MDS is closely related to AML, and roughly one-third of MDS patients eventually progress to AML. Treatment options for MDS are limited, with many patients requiring regular blood transfusions. As in AML, we are focused on subsets of MDS 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 MDS patients have either the RARA or IRF8 super-enhancer, or both.

Ovarian Cancer

Ovarian cancer is the fifth leading cause of cancer-related deaths among women. If caught in the early stages, ovarian cancer is often successfully treated with surgery, chemotherapy and radiation. Common symptoms of ovarian cancer include bloating, abdominal pain, trouble eating or feeling full quickly, and urinary symptoms. These symptoms are often attributed to other causes, however, meaning many women are not diagnosed until after the cancer has begun to spread and becomes markedly more difficult to treat. Chemotherapy remains the standard of care. Despite the emergence of some targeted therapies for ovarian cancer, there remains a need for better treatment options.

Breast Cancer

Breast cancer is the most common type of cancer in women, and 80 percent of breast cancer patients have a form of the disease known as hormone receptor-positive, or HR-positive, breast cancer. If caught early, the outlook for patients with HR-positive breast cancer is generally good. For patients with metastatic HR-positive breast cancer, however, there remains a need for better treatments. Hormone-based therapies, together with a new class of drugs known as CDK4/6 inhibitors, are the standard of care for these patients. Despite the success of these therapies, patients eventually relapse and second-line therapies have limited efficacy.

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