This centre will become the go-to place for researching rare respiratory diseases – which affect both quality of life and life expectancy.  

With the development of a nationwide biobank, new models of disease and early clinical studies, the team hopes to pioneer much-needed therapies for people with rare respiratory diseases. They also hope to improve awareness of what it’s like to live with these conditions, and of available resources that could improve quality of life, by working closely with patient communities.

Lead PI:

Professor Kev Dhaliwal (University of Edinburgh) 

Led by:

University of Edinburgh, University of Nottingham, University of Southampton, University of Cambridge, University College London, University of Dundee 

With co-investigators at:

Action for Pulmonary Fibrosis, University of Edinburgh, University of British Columbia, PCD Support, University of Cambridge, Royal Brompton Hospital, Childhood Interstitial Lung Disease, PCD Research, Nucleic Acid Therapy Accelerator, Wythenshawe Hospital, Leeds General Infirmary, University of Nottingham, LAM Action 

Investment:

£9.4 million  

Almost all kidney failure in children is caused by rare kidney diseases – but a disconnected translational pathway means new treatments don’t often reach those who need it.  

This centre aims to bring transformational change to rare kidney disease research. Its ambitious goals include a clinical trial for a treatment that could cut kidney failure by a third, a new national biobank, and a collaboration to bring all affected children in the UK into the same research network.  

Initially focusing on childhood kidney diseases, the centre later hopes to connect with adult conditions and act as a model for other rare diseases.  

Lead PI:

Dr Louise Oni (University of Liverpool)

Led by:

University of Cambridge, University of Bristol, University College London, University of Leicester, Bristol Children’s Hospital, Kidney Research UK, London School of Hygiene and Tropical Medicine, The UK Kidney Association, patient representatives  

Investment:

£9.4 million

We’re on the cusp of developing urgently needed treatments for mitochondrial diseases – life-limiting genetic conditions that affect 1 in 5,000 people.  

Harnessing recent breakthroughs in our understanding of these conditions, this centre will develop a platform to fast-track new treatments and biomarkers into clinical trials. The team hopes to provide proof-of-principle for 6 new advanced therapies within 5 years.  

Lead PI:

Professor Patrick Chinnery (University of Cambridge) 

Led by:

University of Cambridge, The Lily Foundation, University College London, Newcastle University, with support from Muscular Dystrophy UK 

With co-investigators at:

University of Oxford, University of Birmingham, University of Manchester, University College London, University College London Hospitals NHS Foundation Trust, Great Ormond Street Hospital, Newcastle University, Moorfields Eye Hospital, Cambridge University Hospitals NHS Trust, University of Cambridge 

Investment:

£7.5 million  

Clinical trials represent a major challenge for rare disease translational research. Many fail due to challenges in set-up and delivery, and in the recruitment of small, dispersed patient populations.  

This centre aims to boost the capacity and efficiency of rare disease trials across the UK – including facilitating equitable recruitment, streamlining administrative hurdles and offering a ‘one-stop shop’ for trial design and support.  

Ultimately, the team hopes to help speed up the time it takes for rare disease treatments to reach those who need them.  

Lead PI:

Professor David Jones (Newcastle University) 

Led by:

University of Birmingham, Newcastle University, Queen’s University Belfast 

With co-investigators at:

University of Birmingham, NI Paediatric Clinical Lead for Rare Disease, Chair, Northern Ireland Rare Disease Partnership, Patient Client Council Northern Ireland, Newcastle University, Alström Syndrome UK 

Investment:

£12 million