A young boy with blond hair smiling and reaching out towards colorful confetti hanging in the air, standing near a window with sunlight shining through.

Mission

Sometimes safe and effective treatments for very rare diseases make it through clinical trials and are approved, but the costs are too high and resulting revenues are too low for many companies to make them commercially available to such small patient populations on an ongoing basis.

We founded Orphan Therapies to offer access to these therapies via a unique, non-profit biotech model.

We provide commercial access to approved treatments for very rare conditions