Laboratory news
MRC launches two £50m centres for developing cutting-edge gene therapies
Jan 21 2025
The Medical Research Council (MRC) is launching its first two ‘Centres of Research Excellence’ ─ MRC CoRE ─ which will endeavour to develop transformative advanced therapeutics for currently untreatable diseases and receive up to £50 million each over the next 14 years.
The centres will seek to build upon recent progress made in genomics with advances in genome editing and other gene therapies now making it possible to develop treatments for previously incurable and chronic conditions, such as:
- heart disease
- neurodegenerative conditions like Huntington’s disease
- genetic causes of blindness
- rare genetic diseases affecting children
One centre will be co-funded with the British Heart Foundation (BHF) and called the MRC/BHF CoRE in Advanced Cardiac Therapies. Its focus will be on developing gene therapies for heart disease.
The second centre will be called the MRC CoRE in Therapeutic Genomics and aim to make rare genetic disorders treatable by enabling the affordable production of cutting-edge gene therapies.
The MRC’s CoRE funding model will seek to be transformative in biomedical and health research by aiding discovery of approaches for prevention, early detection, diagnosis, and treatment of diseases through a collaborative approach to research with team members added to the team from wherever they are geographically based.
“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,” said Professor Patrick Chinnery, Executive Chair of MRC.
“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.
“We have seen the first green shoots of how advanced gene therapies could transform medicine, such as the mRNA Covid vaccines, and the approvals for gene-editing therapy that cures blood disorder thalassaemia (Casgevy).
“These two CoREs aim to bring these burgeoning technologies to mass fruition to treat many devastating diseases which will also lead to economic growth,” he said.
MRC/BHF CoRE in Advanced Cardiac Therapies
The MRC/BHF CoRE in Advanced Cardiac Therapies aims to develop the first therapies to stimulate heart repair and regeneration in patients following a heart attack and in those with established heart failure, for which there are currently limited effective treatments.
The researchers will aim to discover and target key processes within the heart tissue, which can stimulate the proliferation of heart muscle cells, encourage the growth of new blood vessels, and counteract the formation of scars. Many of these regenerative processes have been identified as occurring naturally in the hearts of other animals, including salamanders and fish, and even in human infants.
The centre aims to develop the first therapies by using therapies based on nucleic acids, the building blocks of our genetic material DNA and ribonucleic acid (RNA). These will include messenger RNA (mRNA), similar to the cutting-edge techniques in the COVID-19 vaccines, and small regulatory RNAs. These will be identified through systematic, high throughput genetic screening.
“There is a tremendous need for new therapies for heart failure and we’re now at an exciting moment when the technologies have really progressed to an extent where we can realistically start to develop gene therapies. This could be transformational for heart disease treatment,” said Professor Mauro Giacca, director of the MRC/BHF CoRE in Advanced Cardiac Therapies, from King’s College London.
MRC CoRE in Therapeutic Genomics
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.
They aim 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
- severe neurological disorders such as Huntington’s disease
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.
“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,” said Professor Stephan Sanders, director of the new MRC CoRE in Therapeutic Genomics, from the University of Oxford.
Working with UK and international partners
The centre will work with UK and international partners, including:
- Newcastle University
- University College, London
- Karolinska Institute, Sweden
- University of California ─ Innovative Genomics Institute at UC Berkeley and UC San Francisco in the US
To ensure that laboratory work translates into patient benefit, the MRC CoRE in Therapeutic Genomics will also work in partnership with:
- patient groups
- clinicians
- international consortia (N=1 Collaborative)
- industry ─ Danaher, Molecular Devices, IDT, Intellia, Bexorg, La Jolla Labs, the Jackson Laboratory and EveryONE Medicines
- UK infrastructure ─ Oxford-Harrington Rare Disease Centre, Rare Therapies Launch Pad, Genomics England and the Nucleic Acid Therapy Accelerator
—Additional reporting by Alan Booth
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