Led by an experienced team

Management team

We have built a management team with experience in all aspects of biopharmaceutical development and commercialization to drive the company forward.

Board of directors

Our Board provides us with strategic guidance as we advance our mission of making a meaningful difference in the lives of patients with rare genetic diseases.

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Partnerships Card


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Ultra Committed Card

We are Ultra-Committed

Our mission is to transform as many lives of people living with rare diseases as possible.

Learn about our passion