New centre to accelerate gene therapies for heart disease
A new £50m centre is set to develop the first therapies to stimulate heart repair and regeneration in patients with heart disease.
Professor Baker and lab members involved in cardiac regeneration research (L–R) Dr Abdelaziz Beqqali, Dr Matthew Bennett, Professor Andrew Baker, Áine Kelly.
The project, co-funded by the Medical Research Council (MRC) and British Heart Foundation (BHF), builds on the huge progress made in genomics – which allows the genetic basis of many diseases and processes to be identified – and advances in genome editing and other gene therapies.
The MRC/BHF Centre of Research Excellence in Advanced Cardiac Therapies (REACT), co-led by the Universities of Edinburgh, Oxford and King’s College London, is one of two Centres of Research Excellence to each receive up to £50 million over 14 years.
Tissue repair
Heart attacks are the main cause of heart failure, causing the heart to lose muscle because of an interruption of the heart’s blood supply.
Innovative therapies are needed that stimulate formation of new heart tissue, to compensate for any damage or loss following heart failure.
Scientists at the Centre 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 which can reawaken these regenerative processes within the cells of damaged human hearts.
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.
Professor Mauro Giacca
Director of the MRC/BHF Centre of Research Excellence in Advanced Cardiac Therapies, from King’s College London
Cell function
The team will use 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.
The project will use viral and non-viral based technologies to deliver these therapeutic DNAs and RNAs directly into heart cells. Once inside, they will alter the cell’s functions, for example to switch something on or off, or to make a protein.
Industry support
The researchers – supported by Edinburgh Innovations, the University’s commercialisation service – will work closely with the Cell and Gene Therapy Catapult, an independent innovation and technology organisation committed to the advancement of cell and gene therapies, and with industry, including AstraZeneca, AskBio and Batavia Biosciences, to collaborate on tasks such as screening libraries for therapy targets and accessing gene therapy delivery technologies.
They will also work with Syncona, a large venture capital firm in London, to drive further investment and progress toward application of the technology in patients.
We’re building an advanced therapy ecosystem to drive translation from pre-clinical into clinical trials all in one place. Working with industry and venture capital we will also train the next generation of scientists in how to get therapies out of the lab and into clinical practice.
Professor Andrew Baker
Co-director of the MRC/BHF Centre of Research Excellence in Advanced Cardiac Therapies, from the University of Edinburgh
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.
Professor Patrick Chinnery
MRC Executive Chair
The other centre announced by the MRC will be the MRC Centre of Research Excellence in Therapeutic Genomics, which aims to make rare genetic disorders treatable by enabling the mass production of affordable cutting-edge gene therapies.
