©
Dan Burn-Forti
Oxford University researchers are to lead and co-lead two MRC research centres, which are being launched to develop new advanced treatments for currently untreatable diseases.
The Medical Research Council (MRC), part of UKRI, is launching its first two Centres of Research Excellence (CoRE), which will develop transformative new advanced therapeutics for currently untreatable diseases. Oxford is leading one of these Centres and co-leading the other. Together, these international collaborations will receive up to £50 million each over 14 years.
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.
Professor Sarah Wordsworth, Senior Health Economist in Oxford Population Health, and MRC CoRE in Therapeutic Genomics Co-Investigator, commented ‘This study will enable us to examine the costs and consequences of using advanced genomic technologies such as CRISPR gene editing to treat rare genetic disorders in the eye, blood and brain. We will be working closely with healthcare regulators and reimbursement agencies to examine the opportunities and challenges of generating health economic evidence for the use of gene therapies in the NHS and other healthcare systems. This prestigious MRC CoRE funding adds to the department’s long-term research programme on health economics and genomics.’
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. Nicky Whiffin, Associate Professor and Sir Henry Dale Fellow at the Big Data Institute, will lead work to identify for which patients and which genes we can develop genetic therapies.
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.