An international team of researchers has won £30 million for its work in developing a potential cure for inherited heart muscle conditions in young people.
CureHeart was selected as the winner of the British Heart Foundation’s (BHF) Big Beat Challenge, which grants a £30 million non-commercial grant to the winner.
CureHeart is using gene therapy technologies to edit or silence the faulty genes that cause inherited heart muscle diseases.
The team, which is made up of scientists from the UK, US and Singapore, was selected by an International Advisory Panel chaired by Professor Sir Patrick Vallance, Chief Scientific Advisor to the UK Government.
It’s thought that around 12 people under the age of 35 die of an undiagnosed heart condition in the UK every week, according to the BHF. Also known as genetic cardiomyopathies, they often go undiagnosed and are a major cause of heart transplants due to how current treatments do not prevent the condition from progressing.
It’s estimated that one in 250 people worldwide are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children.
CureHeart is using the gene editing technology CRISPR to deploy a technique called base and prime editing in the heart for the first time. This approach uses molecules that can rewrite the single mutations that are buried within the DNA of heart cells in people with genetic cardiomyopathies.
The team is aiming to edit or silence faulty genes that produce abnormal proteins in the heart through re-writing the single spelling mistakes or switching off the entire copy of the faulty gene.
More so, to rectify genes not producing enough protein for the heart muscle, the team aims to increase the production of healthy heart muscle proteins by using genetic tools to correct the function of the faulty copy of the gene or to stimulate the normal copy of the gene.
So far, CureHeart has only tested its approach in animal models but believes therapies could be delivered through the arm to stop disease progression and potentially cure those already living with genetic cardiomyopathies. It could also be used to prevent the disease developing in family members who carry a faulty gene but have not yet developed the condition.
BHF Professor Hugh Watkins, from the Radcliffe Department of Medicine at the University of Oxford and lead investigator of CureHeart, said: “This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure and potential need for a heart transplant. After 30 years of research, we have discovered many of the genes and specific genetic faults responsible for different cardiomyopathies, and how they work. We believe that we will have a gene therapy ready to start testing in clinical trials in the next five years.
“The £30 million from the BHF’s Big Beat Challenge will give us the platform to turbo-charge our progress in finding a cure so the next generation of children diagnosed with genetic cardiomyopathies can live long, happy and productive lives.”
Dr Christine Seidman at Harvard University and co-lead of CureHeart, added: “Acting on our mission will be a truly global effort. We’ve brought in pioneers in new, ultra-precise gene editing, and experts with the techniques to ensure we get our genetic tools straight into the heart safely. It’s because of our world-leading team from three different continents that our initial dream should become reality.”