Thinking Differently About Rare Disease Therapies

By Ben Comer, Chief Editor, Life Science Leader

In the 1990s, before large swaths of the biopharmaceutical industry turned toward rare disease drug development, Dr. Emil Kakkis, an assistant professor at Harbor-UCLA, began developing an enzyme to treat an exceedingly rare disease, known as mucopolysaccharidosis, or MPS I. It was the beginning of a journey that would ultimately lead to the founding of a rare and ultra-rare disease company — Ultragenyx — and the creation of a nonprofit patient advocacy organization — the EveryLife Foundation for Rare Diseases.