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British Heart Foundation
This is for their work on an injectable cure for inherited heart muscle conditions that can kill young people in the prime of their lives.
The team of world-leading scientists from the UK, US and Singapore were given £30 million of BHF research funding last year to boost their efforts to develop the first cures for inherited heart muscle diseases.
They are looking to harness pioneering revolutionary and ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these deadly conditions. The project, called CureHeart, hopes to have a treatment available within a few years.
The British Heart Foundation’s Heart Hero Awards recognise the awe-inspiring people who have gone the extra mile to help those with a heart and circulatory disease. This year‘s Research Story of the Year Award highlights three innovative BHF-funded research projects that have made a huge impact. The public can vote for their favourite on the BHF website until the 20th August, and the winner will be announced at an awards ceremony on Wednesday 6th December.
Inherited heart muscle diseases can cause the heart to stop suddenly or cause progressive heart failure in young people. Every week in the UK, 12 people under the age of 35 die of an undiagnosed heart condition, very often caused by one of these inherited heart muscle diseases, also known as genetic cardiomyopathies.
It’s estimated that one in 250 people worldwide – around 260,000 people in the UK – are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. In many cases, multiple members of the same family will develop heart failure, need a heart transplant, or are lost to sudden cardiac death at a young age.
The team will take the revolutionary gene-editing technology of CRISPR to the next level by deploying ultra-precise techniques, called base and prime editing, in the heart for the first time. These ground-breaking approaches use ingenious molecules that act like tiny pencils to rewrite the single mutations that are buried within the DNA of heart cells in people with genetic cardiomyopathies.
The team have already proven their concept in animals with cardiomyopathies and in human cells. They believe the therapies could be delivered through an injection in the arm that would stop 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.
Professor Watkins 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.
"My team and I are very proud to be shortlisted for the BHF’s Heart Hero Awards. It is an amazing recognition of all the work we have done to get to this point. This project would not be happening without the support the BHF has given us and we think it will go a long way to finally being able to treat these devastating conditions."