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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 or growth hormone deficiency.

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.

Partnering opportunities we are looking for:

  • Monogenetic rare diseases

Example partnerships

Sarepta therapeutics – licensing agreement to launch and commercialize SRP-9001, Sarepta’s investigational micro-dystrophin gene therapy for DMD outside the US

PTC and SMA foundation – collaboration to explore oral therapies for SMA

Your dedicated partnering leads in Rare Diseases


Tom Zioncheck, Head of Neuroscience, Ophthalmology and Rare Diseases, Pharma Partnering


J.C. Lopez, Search & Evaluation Lead Neuroscience, Ophthalmology & Rare Diseases, Pharma Partnering