When a high school athlete dies from a heart attack or a young woman needs a heart transplant, it is often because they inherited a DNA mutation that causes heart muscle disease. Today, in the biggest research grant ever from the research charity, the British Heart Foundation announced an award of £30 million ($36 million) over 5 years to an international team to develop gene-editing treatments for these deadly diseases.
Called CureHeart, the project bested three others shortlisted by the Big Beat Challenge, a competition launched in 2019 to fund transformative heart disease research. Advances in gene editing, particularly the method called CRISPR, “really provide opportunities to combat … cardiovascular disease differently,” said British Heart Foundation Chief Executive Charmaine Griffiths at a press briefing.
The conditions targeted by the award are known as genetic cardiomyopathies. The disorders affect one in every 250 people, putting them—and their relatives who inherit the same mutation—at risk for heart attacks and heart failure. “Cardiomyopathies truly haunt families,” often for generations, says Hugh Watkins, a cardiologist at the University of Oxford and leader of CureHeart.
Researchers know many of the mutations behind these diseases, suggesting one-time injections of gene treatments could cure them. But CureHeart scientists face major challenges, Watkins says. Some defective muscle heart genes are very large, making it impossible to use a virus to ferry in a good copy. In addition, heart researchers may need to correct only the bad copy of a gene and leave a healthy copy untouched, which rules out the classic CRISPR tool that edits by cutting both DNA strands.
To sidestep such issues, CureHeart will deploy twists on CRISPR that repair tiny, single-base errors in just one copy of a gene, as well as strategies that alter the regulatory DNA that controls the gene. “Silencing will be easier,” says co-leader Christine Seidman, a cardiovascular genetics researcher at Brigham and Women’s Hospital and Harvard Medical School. Boosting production of a large protein, however, “will take a lot more molecular trickery.”
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Seidman says the team is encouraged by a recent success at using DNA base editing to repair heart cells and other kinds in mice with progeria, a disease that causes children to age rapidly. Harvard chemist David Liu, whose lab developed base editing, is part of CureHeart. He’s joined by muscle gene-editing and heart disease experts in the United States, United Kingdom, and Singapore.
The plan is to develop four or five approaches aimed at a specific type of mutation and “derisk” them to the point where biotech companies will pick up the approaches and conduct clinical trials, Watkins says. He says the hope is to have “somewhere between one and a few” potential therapies ready for clinical testing within 5 years.
The British Heart Foundation isn’t the only U.K. charity to fund large teams to take on major research challenges. Since 2015, Cancer Research UK has been using this approach to tackle daunting problems in cancer. In June, the group made its third round of $100 million in awards to four teams.