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 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.
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
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.
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.
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.
Related stories
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News releasesFirst-of-a-kind plan announced to get more children access to cutting-edge, proven gene therapy treatments for rare diseases
Read more: First-of-a-kind plan announced to get more children access to cutting-edge, proven gene therapy treatments for rare diseases -
News releasesLifeArc funds pivotal clinical trial to treat an ultra-rare genetic disease with possible link to leader of the mutiny on the Bounty in 1789
Read more: LifeArc funds pivotal clinical trial to treat an ultra-rare genetic disease with possible link to leader of the mutiny on the Bounty in 1789 -
Articles & blogs6 challenges in rare disease research and how we can overcome them
Read more: 6 challenges in rare disease research and how we can overcome them
Got a question for the Rare Disease team?
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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