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A rare approach
to therapeutics
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.
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.
Science & Pipeline
Our expertise, elegant solutions and transparent data narratives have produced a late-stage clinical pipeline with two partnered commercial products.