We’re focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. Our most advanced drug candidate is a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to our active late-stage clinical pipeline, we have multiple novel, investigational therapies in clinical and preclinical development.
We’re a company that cares about our work, each other, and the people who are counting on us the most. We’re inspired to think big and welcome the different perspectives and backgrounds needed to deliver extraordinary results.
healthcare, biotechnology, life sciences, biopharmaceuticals, rare genetic diseases, cellular metabolism
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