Unimagined therapies. Undreamed outcomes.

Despite science’s best efforts, treatments for rare diseases have greatly lagged behind patient needs. It’s time to tackle the undone, the too difficult, and the inaccessible, and change the odds for patients living with rare diseases. Not incrementally — but profoundly.

Breaking news. Breaking conventions.

Our goal is to do more than make headlines. It’s to completely change the narrative for patients with rare diseases.

Proof that the odds are improving.

A clinical trial establishes proof of concept for our anti-HPA-1a monoclonal antibody approach for the prevention of FNAIT.
“Our proven team of innovators is advancing multiple clinical and preclinical programs, charting new paths forward that others haven’t tried. We're also searching the globe to find new targets, product candidates, and partners to match our ambitions.”
—Steve Uden, M.D., Co-Founder and CEO