Recent genetic discoveries and increasing sophistication of gene editing technologies have together aligned to produce a transformative moment in the scientific approach to coronary artery disease.
Genetic studies have revealed naturally occurring gene variants that dramatically lower some individuals’ lifetime risk of coronary artery disease. Gene editing offers the transformational possibility of conferring these same protective variants to adults at risk of the disease.
Overview of Gene Editing
In recent years, gene editing has demonstrated incredible potential to drive new therapeutic breakthroughs to treat disease. Using gene editing, Verve aims to develop therapies, administered as a single dose, that mimic naturally protective variants to confer lifelong protection against coronary artery disease in adults.
Variants in a number of genes have been found to be associated with reduction of risk of heart disease. For example, individuals who have variants that disrupt the PCSK9 gene — which acts to inhibit the removal of cholesterol from the blood — have lower levels of cholesterol, are resistant to heart attack, and are otherwise healthy.
Heterozygous null mutations in the PCSK9 gene, occurring in about 1 in 40 individuals of African ancestry, leads to lifelong lower LDL (low-density lipoprotein) cholesterol and 80% lower risk of heart attack.
Our Therapeutic Vision
Verve has built a stepwise clinical development strategy that first takes aim at adults with life-threatening coronary artery disease and high unmet medical need. As we establish safety and efficacy, we will widen our clinical focus to include progressively larger populations at risk of coronary artery disease.
Explore Verve’s clinical development plan:
STEP 1: Verve’s initial focus will be on adults with established cardiovascular disease who are most in need of new therapeutic approaches.
STEP 2: Verve will then seek to treat adults at high genetic risk for coronary artery disease.
STEP 3: Verve will then offer this therapy to all adults at risk of coronary artery disease in the general population.
Our Ethical Commitment
As we advance in our mission to develop therapies for adults at risk of heart disease, Verve will work in a manner that is consistent with the ethical and scientific frameworks set forth by leading professional societies, regulators, and biomedical ethicists.
All of the therapeutics to be developed by Verve involve making edits in adult (somatic) cells, which are not passed down to offspring. We will not edit embryos, sperm cells, or egg cells.
Safety is paramount for Verve. We will be implementing a rigorous safety protocol with the best available technology for detecting off-target effects. Verve has one of the world-leading experts on gene editing safety on our team, and our clinical development plan will proceed responsibly and transparently.