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King's leads £50m centre to develop cutting-edge RNA therapies for heart disease

11 December 2024

King’s scientists will lead an ambitious centre to develop transformative new advanced therapeutics for heart disease.

The Medical Research Council (MRC) has launched its first two Centres of Research Excellence (CoRE), aiming to develop advanced therapeutics for currently untreatable disease. The new Centres will receive up to £50 million each over 14 years.

The MRC/BHF Centre of Research Excellence in Advanced Cardiac Therapies (REACT), will be co-funded with the British Heart Foundation (BHF) and will focus on developing genetic therapies for heart disease.

Professor Mauro Giacca, Head of the School of Cardiovascular and Metabolic Medicine & Sciences, will lead the centre which 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.

Heart failure affects almost 1 million people in the UK and more than 65 million people around the world. Heart attacks are the main cause of heart failure because they cause loss of the heart muscle due to an interruption of the heart’s blood supply.

To treat heart failure, innovative therapies are needed that stimulate formation of new heart tissue, to compensate for that which is damaged or lost.

The researchers, which include academics from the University of Edinburgh and the University of Oxford, 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 which can reawaken these regenerative processes within the cells of damaged human hearts.

They plan to do this using therapies based on nucleic acids – the building blocks of our genetic material DNA and RNA. These will include 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.

The project will use viral and non-viral based technologies to deliver these therapeutic DNAs and RNAs into the cells of the heart. There they will alter the cell’s functions, for example to switch a function on or off, or to stimulate cardiac cell regeneration.

The goal is to bring one or more novel advanced therapies for heart failure to be ready for clinical trials in the first seven years of the programme.

> 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.

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> Professor Mauro Giacca, Director of the MRC/BHF Centre of Research Excellence in Advanced Cardiac Therapies

He added: "I am truly excited to start our activities. This is a very exciting moment in medicine, as the time is now mature to develop a completely new category of medicines for the heart, based on RNAs and genes. This could be transformational for heart disease treatment.”

The researchers will work closely with the Cell and Gene Therapy Catapult and with industry partners, including AstraZeneca, AskBio and Batavia Biosciences, to collaborate on tasks, such as screening libraries for therapy targets and accessing gene therapy delivery technologies and with Syncona, a large venture capital firm in London, to drive further investment and progress toward application in patients.

The other centre, called the MRC Centre of Research Excellence in Therapeutic Genomics, is led by the University of Oxford and aims to make rare genetic disorders treatable by enabling the mass production of affordable cutting-edge gene therapies.

The MRC’s new CoRE funding model aims to transform biomedical and health research by revolutionising approaches to prevention, early detection, diagnosis, and treatment of diseases by bringing together the very best researchers to tackle the challenge, wherever they are based. In addition, the Centres will be beacons of excellence driving positive changes in research culture, and in training the next generation of pioneers in the field.

> I’m absolutely delighted that our King’s team led by Mauro Giacca will be driving forward this ambitious MRC/BHF-funded CoRE to revolutionise treatment for patients with heart failure. The award builds upon the interdisciplinary critical mass established in the Faculty as well as substantial investment in advanced therapies research and infrastructure over many years.

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> Professor Ajay Shah, Executive Dean of the Faculty of Life Sciences & Medicine

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.”

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