From research to real-world impact
Patients get access to new and better treatments when science, funding and infrastructure come together to deliver medical breakthroughs. Our Centre is deliberately designed to drive faster scientific progress and tackle key gaps and barriers in the journey from research to real-world impact.
An opportunity to transform outcomes
Between advances in genomics and diagnostics and improvements in rare disease research infrastructure, we are genuinely close to transforming outcomes for patients who have waited far too long for effective solutions.
New approaches to clinical trial design are making it possible to generate meaningful evidence in small populations. Regulatory frameworks are evolving to better reflect the realities of rare disease drug and novel therapeutic development. Innovative funding and partnership models are opening new pathways to bring therapies to the patients who need them.
Rare respiratory diseases used to exist in the margins. They were too niche to attract sustained investment, too complex to solve in isolation, and too invisible to the systems designed to support patients. Our Centre is ready to change that.
One translational mission, five focused work packages
Our LifeArc Centre is working to create a future where people with rare respiratory diseases live longer, healthier lives. But the reality is that rare diseases are often neglected and lung research – as a whole – currently receives less than 2% of public health funding. Here’s how we are planning to address the gaps in diagnosis, treatment, resources, training, capacity and awareness that are creating barriers to better health and outcomes for people with rare lung conditions.
“By placing patient voices at the heart of its work and driving system-wide innovation, the Centre has the potential to become a national and global leader in rare respiratory disease.”
Prioritising patients’ needs
We want our work to have a positive, meaningful impact on patients’ lives. We are working closely with patients, carers and patient-advocacy groups to help ensure that our activities reflect their needs and priorities.
Developing national resources
We are establishing new UK-wide resources to facilitate research, diagnosis and clinical trials. Specialised clinical teams will collect samples and data and invite patients to pre-register for trials, which will help to power cutting-edge research and innovation and speed up the delivery of new treatments.
Modelling diseases
We are building laboratory models that mimic the human lung. By using cutting-edge techniques to understand how and why these diseases develop, we are gaining the insights needed to develop and test life-changing treatments.
Discovering therapeutics
We are accelerating the development of effective therapies. We are screening existing medicines to see if they could be used for rare respiratory diseases, and developing advanced genetic therapies to target root causes.
Building capacity and collaboration
We are fostering collaboration and building capacity in the rare disease space. We are driving progress by sharing research expertise, training the next generation of clinical experts, and working closely with patients, clinicians, industry and policymakers to raise awareness and drive change.