Translational Challenge
Rare Disease
Our vision is an ecosystem working together to remove barriers and get tests and treatments to rare disease patients faster.
There are more than 6,000 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.
3.5 million
people in the UK are affected by rare diseases
£30 million
committed to rare diseases by us since 2019
What do we want to achieve?
We’re focused on tackling the major obstacles facing rare disease translational science.
How you can work with us
We partner with and fund the work of academics, charities, healthcare professional and industry on:
Discoveries where our translational expertise can take your research further
Infrastructure and infostructures for collaboration
New therapies and repurposed therapeutics
Commercial, legal and regulatory process to protect your intellectual property
Featured partnerships and resources
Rare Disease Clinical Trials Programme
This programme is an exciting opportunity for researchers working on any rare disease to apply for funding, alongside advice and support from our internal teams, to progress their innovations through clinical trials.
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.
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.
Who to contact
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Amit Nathwani
Read bio: Amit Nathwani
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Rachael Brooker
Read bio: Rachael Brooker
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Paula Kirby
Read bio: Paula Kirby
Related stories
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Articles, News releasesLifeArc joins the REMEDi4ALL Consortium, a pioneering global platform shaping the future of drug repurposing
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News releases£1 million partnership aims to develop new treatments for congenital muscular dystrophy
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News releasesPromising results in clinical trial gives hope to patients with blood disorder
Read more: Promising results in clinical trial gives hope to patients with blood disorder
See our other Translational Challenges
Global Health
Our vision is a world with affordable and accessible solutions to better understand, treat, and prevent infectious diseases.
Motor Neuron Disease
Our vision is a world where motor neuron disease is preventable and treatable.
Chronic Respiratory Infection
Our vision is improved quality of life for those living with cystic fibrosis and bronchiectasis.
Childhood Cancer
Our vision is to drive life-changing innovations for children with cancer.
Contact us
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