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

Thomas F. Zioncheck

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

Hannah Williams

Hannah Williams Search & Evaluation Lead Neuroscience, Ophthalmology & Rare Diseases, Pharma Partnering

roche.com partnering

If you have an opportunity, we would like to hear from you.

Contact us

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