Dedicated to people with rare diseases

We’re here to make a positive difference in the lives of patients. We build sincere and trusting relationships based on humility, transparency and a clear desire to listen and to understand their experiences of living with a rare disease.

Accelerating our impact

Building on nearly 15 years of pioneering research in cellular metabolism and leveraging our differentiated translational and clinical capabilities, we are advancing new medicines in the therapeutic areas where we believe our expertise can have the biggest impact: rare diseases.

Diseases are typically considered rare if they affect fewer than 200,000 people in the United States. Many of these diseases are severe or life-threatening, and current treatment options for these disorders are generally limited. Our goal is to develop mechanistically specific, small molecule approaches with the potential to have disease modifying and long-term rather than palliative effects.

Today, our focus is on hemolytic anemias. People living with hemolytic anemias are at risk of both acute symptoms and long-term complications, and the disorders may significantly impact their quality of life and daily functioning. We specifically serve people with the following hemolytic anemias:

Pyruvate Kinase Deficiency

Pyruvate kinase (PK) deficiency is a rare disease caused by an inherited mutation in the PKLR gene, which can cause a deficit in energy within red blood cells.

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Thalassemia is an inherited blood disorder caused by mutations in either alpha- or beta-globin genes. As a result, the body is not able to make enough hemoglobin, an important part of red blood cells.

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Sickle Cell Disease

Although a rare disease in the U.S. and EU, sickle cell disease is one of the most common genetic disorders in the world. It is caused by inherited mutations in the beta-globin gene, leading to sickle-shaped red blood cells that slow or stop the flow of blood.

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Activating a critical pathway

We are changing the way rare diseases are viewed and treated. Building on our pioneering leadership in cellular metabolism, we may be able to address a broad range of hemolytic anemias to develop life-changing therapies for patients.

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We’re looking for energetic colleagues to fuel our mission of creating life-changing therapies for patients with rare diseases. Join us!