Heart disease: £30million grant to develop injectable cure for killer genetic conditions

Professor Hugh Watkins on genetic medicines for heart disease

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Inherited heart muscle diseases, also known as genetic cardiomyopathies, can cause the heart to either stop suddenly or cause progressive failure. Around 260,000 individuals in the UK are affected by genetic cardiomyopathies — and there is a 50 percent chance that they will pass their faulty genes on to the next generation. These conditions can affect people at a relatively young age. In fact, every week in the UK, around 12 people under the age of 35 die of an undiagnosed heart condition. Around half of all heart transplants are required as a result of cardiomyopathy and current treatments are not able to stop the condition from progressing.

It is hoped that the £30million award delivered by the British Heart Foundation’s Big Beat Challenge — one of the largest non-commercial grants ever awarded — will provide hope for families impacted by these killer diseases.

The winning team, CureHeart, is looking to provide the first-ever cures for these inherited conditions through the development of ultra-precise gene therapy technologies that will either edit or silence the faulty genes responsible for these deadly heart problems.

CureHeart is made up of world-leading scientists from the UK, the US and Singapore.

They were selected to win the Big Beat Challenge by an international advisory panel chaired by the UK Government’s Chief Scientific Adviser, Professor Sir Patrick Vallance.

Sir Patrick said: “CureHeart was selected in recognition of the boldness of its ambition, the scale of its potential benefits for patients with genetic heart muscle diseases and their families, and the excellence of the international team of participating researchers.”

CureHeart’s lead investigator and cardiologist Professor Hugh Watkins of the University of Oxford said: “This is a once-in-a-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 £30million from the British Heart Foundation’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.”

Fundamentally, the researchers will be using the gene editing tool CRISPR, which allows biologists to make precision cuts on DNA, allowing genes to be inserted, removed or replaced at a desired point in order to edit a genome.

However, CureHeart aims to take CRISPR to the next level by using ultra-precise base and prime editing techniques in the heart for the first time.

They will focus these methods on two areas — first either correcting or switching off faulty genes that result in the production of abnormal proteins in the heart’s pumping machinery.

Meanwhile, in cases where a faulty gene produces insufficient amounts of a protein that is vital to heart function, the team will use their editing tools to either correct the fault or stimulate the normal copy of the gene.

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CureHeart co-leader and cardiovascular geneticist, Dr Christine Seidman of Harvard University, said: “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.”

The researchers have already demonstrated the efficacy of their technique in both animals with cardiomyopathies and human cells in the laboratory.

The ultimate therapy, they added, would likely be delivered by means of an injection to the arm, and could both stop existing heart muscle conditions from spreading — and potentially even reverse them —alongside stopping the disease from developing in patients who carry the faulty genes but have not yet developed the condition.

British Heart Foundation Medical Director Professor Sir Nilesh Samani said: “This is a defining moment for cardiovascular medicine.

“Not only could CureHeart be the creators of the first cure for inherited heart muscle diseases by tackling killer genes that run through famly trees, it could also usher in a new era of precision cardiology.

“Once successful, the same gene editing innovations could be used to treat a whole range of common heart conditions where genetic faults play a major tole.

“This would have a transformational impact and offer hope to the thousands of families worldwide affected by these devastating diseases.”

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