Unlocking potential to create new possibilities

Our mission


To improve the lives of patients with rare diseases and cancer.

Our progress


Driven by a true understanding of the critical nature of our mission, we harness the power of science and meaningful relationships to create new possibilities for patients who need them.

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Discover our revolutionary therapies

We are committed to helping patients with rare diseases and cancer.

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Learn about our valuable opportunities for investors

We identify and develop therapies with the greatest unrealized, long-term potential.
Visit Investor Center

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See how we are working with you

We know the value in collaborating with partners, patient communities and healthcare professionals.