Rare Respiratory Disease Industry Accelerator Day 'Getting Cilia Moving' on Tuesday, 1 October 2024, at the Francis Crick Institute, London, UK in association with PCD Research, LifeArc and Weatherden. 

Globally, more than 300 million people are living with rare diseases, of which one in 20 are related to respiratory conditions. While rare on their own, collectively they represent significant social and economic costs. Given more than 95% of rare diseases lack an FDA-approved treatment, rare respiratory diseases represent a phenomenal growth opportunity if we can come together to overcome some of the unique challenges within the rare disease space. With our new LifeArc Centre for Rare Respiratory Diseases, we embody this bold ambition to improve diagnosis and care for patients living with rare respiratory disease and foster innovation for much-needed treatments and cures.

Leading with three disease exemplars, we aim to derisk translation from discovery through to clinical trials. Here, we showcase primary ciliary dyskinesia (PCD), which affects 1:7000 births, and impairs mucociliary clearance leading to decline in lung function similar to cystic fibrosis (CF). Unlike CF, no approved treatments exist for PCD, despite a similar market size and unmet significant need. Together, we aim to change this.

We see PCD as a test case, with wider applicability across rare disease and personalised medicine showing how we can connect all the dots to market. This event looks to leverage intellectual property from cystic fibrosis into PCD, together with a wider conversation on how we can drive transformational change in rare disease from development through to policy and regulation- all to make therapies accessible for the >95% of people with a rare disease who lack them.

With our world-leading lineup of experts in genomics, respiratory medicine, rare disease and genetic therapies together with key UK policymakers and regulators, we look to create a unique event to engage innovators and disruptors in translational research, and leaders in the rare disease pipeline development space across biopharma and the investment sector.

Tickets are available here. 

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