OUR MISSION is to deliver new treatments for the 350 million affected by rare diseases worldwide
OUR GOAL is to deliver 20 drugs in the next 10 years
Worldwide Health Challenge
350 million people across the globe have a rare disease, affecting 1 in 17 people during their life. Rare diseases disproportionately impact children, with a third of affected children dying before their fifth birthday. Patients with rare diseases, and their families, often have a long journey to diagnosis and little hope for a cure. Fewer than 5% of rare diseases have approved treatments. Powerful advances in DNA sequencing, scientific research and drug development will make curing rare diseases a reality.
"Our partnership with Harrington Discovery Institute recognises the combined experience needed in world-class science and drug development if we are to change the rare disease landscape in a meaningful way."
– Sir John Bell, University of Oxford