The Medical Research Council (MRC), part of UK Research and Innovation (UKRI), has launched its Centre of Research Excellence in Therapeutic Genomics (CoRE), which will receive up to £50 million funding over 14 years.

Joining UK and international partners in the Therapeutic Genomics MRC CoRE, Newcastle bring their clinical and research expertise in inherited disorders of the immune system caused by ‘spelling mistakes’ in single genes.

Delivering new treatments

Research at Newcastle has focused on uncovering the genetic origins of inborn errors of immunity. Therapeutic genomics promises to bridge these aims by connecting the genetic diagnosis to a personalised treatment for each affected child.

Sophie Hambleton, Professor of Paediatrics and Immunology at Newcastle University, said: “I am thrilled to be part of this exciting initiative to reimagine the approach to genetic therapy for inherited diseases. 

“Through the MRC CoRE, we can leverage the power of our multi-sector team to build a shared platform for safe development and delivery of these new treatments. 

“Importantly, we can do this on a timescale that is relevant to the many affected children and families who are crying out for better alternatives to current treatments.”

The new MRC CoRE in Therapeutic Genomics aims to transform the diagnosis and treatment of genetic disorders by enabling the mass development of cutting-edge genetic therapies.

The aim is to develop therapies for many devastating genetic disorders that are currently untreatable, such as rare disorders that cause severe seizures in infants and neurodevelopmental delay, certain types of blindness and immune disorders, and severe neurological disorders such as Huntington’s Disease.

Recent breakthroughs in genomics and the first generation of genetic therapies have started to revolutionise the treatment of a few genetic disorders. However, the process to create, test, and approve each new therapy is too slow and expensive to enable treatments to be developed for the thousands of genetic disorders being diagnosed.

To overcome this, the Centre hopes to develop processes to take successful genetic therapies and re-programme them to treat new disorders. It will also use artificial intelligence approaches to enable scientists to process huge amounts of genetic data from patients at previously unimaginable depth.

‘Paradigm shift in knowledge’

Professor Stephan Sanders, Director of the new MRC CoRE in Therapeutic Genomics, from the University of Oxford, said: “Reprogramming genetic therapies has the potential to treat thousands of genetic disorders.

“The new centre will help create a paradigm shift in the knowledge, infrastructure, technology, and industry regulation so that we can make safe and effective patient-customised therapies en masse.”

Researchers will initially focus on developing genetic therapies for disorders of the blood, eye, and brain.

The knowledge gained from treating these disorders will be shared widely, enabling these approaches to be extended to increasingly large numbers of disorders and organs by multiple research groups.