Translational Challenge

Rare Disease

Our vision is an ecosystem working together to remove barriers and get tests and treatments to rare disease patients faster.

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There are more than 8,000 rare diseases and over 95% of them don’t have an approved treatment.

We’re looking at rare conditions collectively and how our experience in science translation can help progress promising discoveries.

We seek to use our expertise and collaborate with others to tackle some of the systematic issues in getting discoveries from the lab to the next phase of development, and ultimately to patients who need them.

What do we want to achieve?


We’re focused on tackling the major obstacles facing rare disease translational science.

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How you can work with us

We partner with and fund the work of academics, charities, healthcare professional and industry on:

Featured partnerships and resources


The LifeArc Philanthropic Fund

Grants and funding for researchers working to advance new treatments and diagnostics for rare diseases.

Joint funding call: repurposing therapies for EB

LifeArc and DEBRA Austria teamed up to fund projects to develop therapeutics for Epidermolysis Bullosa.

Child affected by EB with her father. Photo ©Debra Austria

LifeArc Gene Therapy Innovation Fund

A £5m annual fund to support manufacture of viral vector at the world-class network of Innovation Hubs for Gene Therapies.

LifeArc Translational Centres for Rare Diseases

Our £40 million investment into 4 research centres will unlock new tests, treatments and potentially cures for people living with rare diseases.  

Drug repurposing resources

View our drug repurposing report and toolkit.

Drug repurposing report
Drug repurposing toolkit

How we work with charities

Rare disease charities can access a range of services including technology transfer, IP protection and advice on progressing research toward commercial success.

LifeArc staff working through ideas on Post-It notes

Who to contact

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