A rare approach
to therapeutics
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A rare approach
Who We Are
We are a purpose-driven company focused on bringing life-changing therapeutics to people living with rare diseases.
What We Do
We address the unmet needs of patients living with rare diseases through leading scientific advances and the collective strength of passionate people.
Why We Do It
We are zealous in our pursuit of developing and delivering therapies that address significant unmet needs because patients and their care partners don’t have a moment to lose.
Science & Pipeline
Our expertise, data-driven scientific approach and patient-first perspective has resulted in two commercial products, a late-stage clinical pipeline, and one partnered product.