95%. Of diseases. Have no cure.

I · The Demand — Of ~10,000 known diseases, fewer than 5% have an approved therapy.NIH Office of Rare Diseases Research · FDA Orphan Drug Program

300 million. Patients. Waiting.

The Volume — Approximately 300–400 million people live with a rare disease alone — most without a treatment.WHO · Global Burden of Disease

Demand Wait Build