Monogenetic rare diseases
350 million people live with a rare disease - the vast majority of them are children. Today, only 200 of the 7,000 rare diseases have available treatments for the impacted individuals.
We have a proud history of transforming the lives of those with rare diseases for example, treatments for people with microscopic polyangiitis (MPA), non-Hodgkin’s lymphoma, idiopathic pulmonary fibrosis, haemophilia A, growth hormone deficiency or Spinal Muscular Atrophy.
We are strongly committed to building on this legacy, bringing the full capabilities and know-how of our scientists together to bring new treatments and diagnostic tests for rare diseases to the people living with fatal or life-limiting rare diseases to lead the most fulfilling lives possible.
If you have an opportunity for collaboration or an interesting technology, or if you have an asset in one of our areas of interest, we would like to hear from you.