LifeArc National Translational Platform for Respiratory Rare Disease

There are currently no treatments for most rare lung diseases, and funding for research into rare diseases is limited. Excellent research is happening in the UK, but is occurring in silos. By coming together we will rapidly build infrastructure and expertise that can be shared across lung diseases, and which will be ‘the go to resource’ for researchers and drug companies. Our Centre aims to find potential treatments and cures for respiratory rare disease and to ensure the treatments are ready for clinical trials by demonstrating that they work in laboratory in models that are relevant to patients. We will start with three types of rare lung diseases, primary ciliary dyskinesia, cystic lung disease and COPA syndrome which encompass genetic, immune and inflammatory conditions and span all ages. We will use these three conditions to prove the centre can deliver our specific aims which are: • to bring together patients, carers, scientists and doctors to create a shared vision for all respiratory rare diseases and the Centre, and nurture the next generation of stakeholders • a network that will collect precious samples and information from people with respiratory rare diseases across the UK • to use patient samples to create the tools that scientists can use to test potentially thousands of therapies • to select promising therapies to test in small numbers of patients. This strategy will draw in pharmaceutical industry partners that will be needed to scale up for the types of trials needed to produce effective medicines for respiratory rare diseases