Philadelphia, PA, 12 November 2023 – A South Asian participant in the Genes & Health study run by Queen Mary University of London and Barts Health NHS Trust was one of ten people globally to be enrolled in a phase 1 clinical trial, to evaluate VERVE-101, a single dose, once and done, gene-editing treatment designed to reduce high-cholesterol caused by familial hypercholesterolaemia (FH).
Verve Therapeutics, the biotechnology company running the trial announced initial results from the study today (12 November) at the American Heart Association’s Scientific Sessions conference.
The participants enrolled in the heart-1 phase 1b trial have a heterozygous form of familial hypercholesterolaemia (FH), a genetic condition that leads to very high low-density lipoprotein (LDL) cholesterol levels and, in turn, increases the risk of coronary artery heart disease. The participant involved in the trial was diagnosed with FH by Queen Mary’s Genes & Health, one of the world’s largest community-based genetics studies that aims to improve health outcomes of British South Asians and diversify genetic data. Through a collaborative effort, the patient was referred to Barts Health NHS Trust, who in conjunction with Richmond Pharmacology Ltd (the site where the patient was dosed and clinically managed), provided the opportunity for this patient to enrol in this ground-breaking trial.
FH is one of the most common genetic conditions, affecting around 1 in 300 people globally. However, fewer than 10% of those affected worldwide will ever receive a diagnosis and most remain untreated. Without treatment, 50% of men will have a heart attack before they’re 50 and 30% of women by the age of 60.
The Genes & Health participant who received VERVE-101 said:
“Heart attacks run in my family, which is why I decided to participate in the heart-1 clinical trial...I was afraid that I would be next. That was my motivation for being one of the first in the world to try VERVE-101. While I was only treated a few weeks ago, I’m very optimistic that the medicine will work, and I won’t have to worry about having a heart attack.”
Most studies that report on the prevalence for FH in the general population are from Europe, North America, East Asia and Australia; it is unclear how common this condition is in different parts of the world and in minority ethnic communities. Bangladeshi and Pakistani people are underrepresented in genetic research despite having some of the highest rates of heart disease, diabetes, and poor health in the UK. Genes & Health aims to change that.
Professor David van Heel, Chief Investigator and Joint Lead of Genes & Health said:
“We are very proud to have had a Genes & Health participant involved in this important clinical trial. These results also happen to coincide with the milestone of diagnosing, and initiating treatment, for our 50th Genes & Health volunteer with familial hypercholesterolemia. From the beginning of Genes & Health we wanted to do something about the very high rates of heart disease in our volunteer communities, and it is amazing to be able to do so.”
Genomic testing for FH is one of the priorities of the NHS Genomic Medicine Service. Genes & Health, together with Barts Heart Centre, have developed a pathway for research participants to receive a FH diagnosis and get the right treatment, early. So far, 50 participants have been diagnosed with FH through Genes & Health, a significant achievement for the project.
FH is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol, leading to higher disease-causing LDL-cholesterol in the bloodstream. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Verve Therapeutics have developed a single-dose gene editing medication which targets the affected gene PCSK9 in the liver to reduce LDL-cholesterol.
To see the initial data from the heart-1 study, click here. (link to be inserted on Sunday evening GMT)
NOTES TO EDITORS
Contact
Sophia Prout, Faculty Communications Manager – Medicine and Dentistry, Queen Mary University of London
+44 7718136512
Mehru Raza, Communications & Engagement Manager – Genes & Health, Queen Mary University of London
+44 7583205052
About heart-1
heart-1 is an open-label Phase 1b clinical trial designed to enrol adult patients with heterozygous familial hypercholesterolemia (HeFH) who have established atherosclerotic cardiovascular disease (ASCVD) to evaluate the safety and tolerability of VERVE-101 administration, with additional analyses for pharmacokinetics and reductions in blood PCSK9 protein and low-density lipoprotein cholesterol (LDL-C). The study is run by Verve Therapeutics.
About Richmond Pharmacology Ltd
Richmond Pharmacology, London Bridge, United Kingdom, is as a global leader in conducting gene editing clinical trials, boasting the most extensive experience in dosing patients into these ground-breaking therapies. With a proven track record of success, Richmond continues to play a pivotal role in advancing gene editing research, aiding pharmaceutical and biotechnology companies in paving the way for potential curative treatments for a wide range of debilitating diseases. The company's expertise lies in its meticulous approach to patient safety and its commitment to conducting trials with the utmost rigor and scientific integrity. Richmond's dedication to innovation has propelled it to the forefront of gene editing, positioning it as a driving force in this transformative field of medicine.
About Queen Mary University of London
At Queen Mary University of London, we believe that a diversity of ideas helps us achieve the previously unthinkable.
Throughout our history, we’ve fostered social justice and improved lives through academic excellence. And we continue to live and breathe this spirit today, not because it’s simply ‘the right thing to do’ but for what it helps us achieve and the intellectual brilliance it delivers.
Our reformer heritage informs our conviction that great ideas can and should come from anywhere. It’s an approach that has brought results across the globe, from the communities of east London to the favelas of Rio de Janeiro.
We continue to embrace diversity of thought and opinion in everything we do, in the belief that when views collide, disciplines interact, and perspectives intersect, truly original thought takes form.
About Genes & Health
https://www.genesandhealth.org/
Genes & Health is core-funded by Wellcome, the Medical Research Council, Higher Education Funding Council for England Catalyst, Barts Charity, and Health Data Research UK. Queen Mary’s Genes & Health group are trying to reduce health inequalities by analysing the genetic information of 100,000 Bangladeshi and Pakistani people to improve health outcomes locally and globally.
About Barts Health NHS Trust
https://www.bartshealth.nhs.uk/
With a turnover of £1.7billion and a workforce of around 17,000, Barts Health is a leading healthcare provider in Britain and one of the largest NHS trusts in the country. The Trust’s five hospitals – St Bartholomew’s Hospital in the City, including the Barts Heart Centre, The Royal London Hospital in Whitechapel, Newham Hospital in Plaistow, Whipps Cross Hospital in Leytonstone and Mile End Hospital – deliver high quality compassionate care to the 2.5 million people of east London and beyond.
Method of Research
Randomized controlled/clinical trial
Subject of Research
People
Article Title
A participant from Queen Mary University of London Genes & Health study is the 10th person enrolled in a gene-editing clinical trial for heart disease
Article Publication Date
12-Nov-2023