We are a collection of individuals focused on discovering and developing drugs for patients with rare genetic diseases. We do this by:
Finding the right starting points to target diseases at their source.
Using a combination of a systematic assessment of the genetic disease landscape and informed diligence on the latest research, we find indications with clear mechanisms of pathogenesis that are ripe for translation into disease-modifying drugs.
Building products with world-class R&D personnel.
We employ over 50 scientists and work closely with leading academics to prosecute a wide variety of drug programs at the highest level of scientific rigor. We combine biopharmaceutical veterans with up-and-coming leaders to drive entrepreneurial thinking grounded by experience.
Employing a lean, capital efficient model.
We house each effort in an individual subsidiary company supported by shared central resources. This efficient, de-centralized corporate structure enables focus at level of each disease, but provides diversification and the ability to scale across many opportunities.
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