About Us

Mission & Vision

For more than a decade, our mission has been to create differentiated, small molecule medicines for patients in three focus areas – malignant hematology, solid tumors and rare genetic diseases – based on our unique expertise in cellular metabolism and adjacent areas of biology.

Our Areas of Focus

Hematologic Malignancies

Solid Tumors

Rare Genetic Diseases

Inspired by patients and frustrated by the limitations of conventional approaches to treatment, Agios pioneered a novel path to treating cancer and rare genetic diseases by targeting cellular metabolism.

In our first decade, Agios brought two precision oncology medications from our own labs to patients – TIBSOVO® (ivosidenib tablets) and IDHIFA® (enasidenib). During that same time period, the Agios team discovered and developed six additional investigational new drug (IND) candidates, including the first pyruvate kinase-R (PKR) activator in development as a potential treatment for a rare hemolytic anemia known as pyruvate kinase deficiency.
Agios is leading the way in advancing PKR activation for additional non-malignant hematologic diseases, including thalassemia and sickle cell disease, and the company continues to foster a productive research engine that yields new insights and potential therapeutic approaches.

Now entering our next decade, Agios has a differentiated portfolio of preclinical, clinical and commercial programs, unparalleled expertise in cellular metabolism and a committed team that will continue to drive the company’s mission to make a difference for patients with hematologic malignances, solid tumors and rare genetic diseases.

Agios 2025 Strategic Vision

At the beginning of 2020, we outlined a strategic vision for the company’s exciting next chapter of growth.

As part of this vision, Agios expects to achieve the following milestones by the end of 2025:

  • 4 marketed medicines discovered and developed at Agios
  • Approvals in 8+ indications spanning hematologic malignancies, solid tumors and rare genetic diseases
  • 6+ molecules in the clinic generated by the company’s internal research discovery engine
  • Cash-flow positive within the six-year timeframe