A rare approach
to therapeutics

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Photo of an older man carrying a young girl, helping her to water plants with a watering can.
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Who We Are

We are a nimble rare disease therapeutics company comprised of expert scientists, patient advocates, development strategists, commercialization specialists, and business development gurus with a proven record of bringing new therapies to patients.

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A child sits happily on an examination table looking up at her doctor while her father looks on.

What We Do

We overcome the challenges of traditional drug development by following the data and weaving a compelling regulatory narrative that puts unmet needs of patients front and center.

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.

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Science & Pipeline

Our expertise, elegant solutions and transparent data narratives have produced a late-stage clinical pipeline with two partnered commercial products.