Rare Diseases

Partnering opportunities we are looking for:

  • Monogenetic rare diseases

Example partnerships

350 million people live with a rare disease - the vast majority of them are children. Today, only 200 of the 10,000 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.

Your dedicated partnering leads in Rare Diseases

Tom Zioncheck
Head of Neuroscience, Ophthalmology and Rare Diseases, Pharma Partnering
Hannah Williams
Search & Evaluation Lead Neuroscience, Ophthalmology & Rare Diseases, Pharma Partnering

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.

Contact our Pharma Partnering Team

Interested in partnering with us?

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