Luke Robinson
VP and General Manager, Ireland, UK and the Nordic Countries, BioCryst
Learn how a small biotech built on a big dream considers underserved patient populations at the heart of its innovation.
Although each rare disease may have a small patient population, the aggregate of all rare diseases means that a substantial proportion of people are impacted. Current conservative estimates suggest that 18–30 million people in the EU and 263–446 million people worldwide are affected by rare disease at any point in time.1
Commitment to rare disease advancement
BioCryst, a commercial-stage US biotech with EMEA headquarters in Dublin, is committed to discovering and developing treatments and has spent over three decades working to meet unmet needs in rare disease. Luke Robinson, VP and General Manager for Ireland, UK and Nordic Countries suggests: “Many rare diseases share common challenges, such as difficulty in diagnosis, limited treatment options and a lack of awareness, making the collective experience of people living with these conditions more significant. As a result, rare diseases are a growing area of focus for researchers, policymakers and all those who support the local healthcare infrastructure.”
Delivering targeted solutions for unmet patient need
BioCryst focuses on the development of therapies for rare disease. An example of this is hereditary angioedema (HAE), a potentially life-threatening rare disease characterised by repeated painful and unpredictable swelling attacks.
Rare diseases are a growing area of focus
for researchers, policymakers and all those
who support the local healthcare infrastructure.
Researchers at its Discovery Centre of Excellence use an innovative structure-guided approach to drug design, which involves the use of computational tools and techniques to analyse the molecular structure of disease-causing proteins. By understanding the shape and dynamics of these proteins and essentially how they ‘fit together,’ BioCryst can design small molecules that bind specifically to these targets, either inhibiting their harmful activity or modulating their function in a beneficial way. This approach is particularly useful in the treatment of rare diseases, where targets may be poorly understood or difficult to modulate using traditional drug discovery methods.
Collaboration supporting innovation
Innovation and collaboration in rare disease accelerate the development of new drugs and improve the precision of therapies. They give people living with rare disease a chance of achieving health equity, supporting the better management of their conditions and quality of life in a practical way.
With BioCryst’s approach to innovation, listening, learning and sharing, it has made strides across Europe in working with patient groups, regulators and Health Technology Assessment (HTA) bodies to bring new medicines to those who need them most. “I am proud of our approach and while much has been achieved, I am looking forward to what is yet to come,” concludes Robinson.
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[1] Nguengang Wakap, S., Lambert, D.M., Olry, A. et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet 28, 165–173 (2020).
