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Dan Burn-Forti
The Centres will build on the huge progress that has been made in genomics – allowing the genetic basis of many diseases and processes to be identified – and advances in genome editing and other gene therapies, which have made it possible to develop treatments for previously incurable conditions.
The Centres will take different approaches to translating the advances in genomics into therapies to treat many diseases, such as heart disease, severe immune disorders, genetic causes of blindness, many developmental disorders that affect children, including those that cause severe seizures in babies, and neurodegenerative conditions including Huntingdon’s disease.
The MRC CoRE in Therapeutic Genomics aims to make rare genetic disorders treatable by enabling the mass production of affordable cutting-edge gene therapies.
Nicky Whiffin, Associate Professor and Sir Henry Dale Fellow at the Big Data Institute and Centre for Human Genetics, and MRC CoRE in Therapeutic Genomics Co-Investigator, said: ‘We will harness large-scale data, including genetic data from UK Biobank and Genomics England, to identify for which patients and which genes we can develop genetic therapies. We will also use data from different ‘omics’ technologies to predict new ways in which we can target these genes, hoping to modify how they are expressed (turned on or off) in different tissues.’
Professor Whiffin will also be a co-lead for training and research culture in the CoRE, she said: ‘I am very excited to have this important role in the Centre. We hope to build on foundations of collaboration, open science, and mentorship, while driving positive changes in research culture.’
Recent breakthroughs in genomics and the first generation of genetic therapies have begun 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 aims to develop processes to take successful genetic therapies and reprogramme them to treat new disorders. The new Centre will also use artificial intelligence approaches to enable scientists to process huge amounts of genetic data from patients at previously unimaginable depth.
Professor Stephan Sanders, Director of the new MRC CoRE in Therapeutic Genomics, 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.’
Professor Deborah Gill, Co-Director of the MRC CoRE in Therapeutic Genomics, said: ‘We will also prioritise innovation in research culture, ensuring that science is conducted in an ethical and responsible manner, incorporating feedback from patients and the public, so that the findings are distributed to benefit society. To achieve our vision, we will recruit talented researchers and students and teach them to consider every step of the way from lab to clinic.’
Professor Patrick Chinnery, MRC Executive Chair, said: ‘The MRC CoREs are a new way of funding bold and ambitious science that seeks to advance our ability to understand diseases, diagnose them at an early stage, intervene with new treatments and prevent diseases of the future. They will focus on bringing together the brightest scientists to tackle diseases of major medical importance, so that they will really change the landscape and improve the health of the nation.
‘I am excited to see how the first two centres announced today will transform approaches in advanced therapeutics. We have seen the first green shoots of how advanced gene therapies could transform medicine, such as the mRNA Covid vaccines, or the recent announcement of the NHS approving a gene-editing therapy that could cure blood disorder thalassaemia. These two CoREs aim to bring these burgeoning technologies to mass fruition to treat many devastating diseases which will also lead to economic growth.’
The other centre, called the MRC/BHF CoRE in Advanced Cardiac Therapies, will be co-funded with the British Heart Foundation (BHF) and will focus on developing gene therapies for heart disease.