Corporate Profile

We are a clinical-stage biotechnology company built around a team of seasoned industry experts with a track record of success in discovering, developing, manufacturing, and delivering therapies. We believe we have assembled the best people, partners, and science to achieve our goal of meaningfully improving the lives of patients suffering from severe and rare diseases.

Our portfolio of promising product candidates addresses rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, ophthalmology, and metabolic disorders. Through our extensive network of relationships with academic clinical centers and leaders in industry worldwide, we are uniquely positioned to advance and further accelerate our portfolio, as well as seek new opportunities.

We are drawing on our decades of knowledge and experience with a determination to tackle the undone, the too difficult, the inaccessible — and change the odds for rare disease patients.

Investment Highlights