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
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.
The TIARA trial: Investigating a new treatment for a life-threatening blood disorder
A collaboration with the Aplastic Anaemia Trust to jointly fund a trial testing a cell therapy as a potential new treatment for aplastic anaemia.
The Rare Disease Taskforce Report
This report, developed with Genetic Alliance UK and our Rare Disease Taskforce, gives recommendations to streamline the UK R&D landscape to accelerate diagnoses and access to treatments.
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
-
Amit Nathwani
Read bio: Amit Nathwani
-
Rachael Brooker
Read bio: Rachael Brooker
-
Paula Kirby
Read bio: Paula Kirby
Related stories
-
News releasesBirmingham researchers lead world-first trial for rare liver disease treatment funded by LifeArc
Read more: Birmingham researchers lead world-first trial for rare liver disease treatment funded by LifeArc -
ArticlesLifeArc Pathfinder Award recipients uncover genetic cause of severe nerve condition in children
Read more: LifeArc Pathfinder Award recipients uncover genetic cause of severe nerve condition in children -
ArticlesLifeArc and Genetic Alliance UK launch a new report calling for the acceleration of research and development (R&D) for rare diseases
Read more: LifeArc and Genetic Alliance UK launch a new report calling for the acceleration of research and development (R&D) for rare diseases
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 & Rare Respiratory Disease
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
In submitting your personal data via this form, you consent to being contacted via the details provided so that your enquiry can be responded to. If you would like your data to be removed, please email dataprivacy@lifearc.org.
Please see our Privacy Policy in relation to the personal data you submit to us through this page.
This site is protected by reCAPTCHA and the Google privacy policy and terms apply.