Recent genetic discoveries and increasing sophistication of gene editing technologies have together aligned to produce a transformative moment in the scientific approach to cardiovascular disease (CVD).
Genetic studies have revealed naturally occurring gene variants that dramatically lower some individuals’ lifetime risk of atherosclerotic cardiovascular disease (ASCVD) and protect against heart attack. Our programs are designed to mimic these natural disease resistance mutations and turn off specific genes in order to lower blood lipids through a single-course therapy, thereby reducing the risk of ASCVD.
Overview of Gene Editing
In recent years, gene editing has demonstrated incredible potential to drive new therapeutic breakthroughs to treat disease. Using gene editing, we aim to disrupt the chronic care model for CVD by providing a new therapeutic approach with single-course in vivo liver-directed gene editing treatments focused on addressing the root causes of this highly prevalent and life-threatening disease.
ASCVD Causal Factors and Gene Targets
High cumulative life-long exposure to blood cholesterol, which is carried in LDL, triglyceride-rich lipoprotein (TRL) or lipoprotein(a) (Lp(a)), is a root cause of ASCVD. Each of these three lipoproteins represents an independent pathway of risk for ASCVD. A gene editing treatment targeting each pathway could provide additive benefits for the treatment of ASCVD.
Our most advanced gene editing programs targeting two independent pathways controlling blood lipids implicated in ASCVD risk – PCSK9 and ANGPTL3. We also plan to expand beyond PCSK9 and ANGPTL3 to develop a suite of single-course gene editing medicines that could comprehensively and robustly address other independent causes of ASCVD.
Our Ethical Commitment
As we advance in our mission of transforming treatment for patients with cardiovascular disease from chronic management to single-course gene editing medicines, we 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 single-course gene editing medicines to be developed by Verve involve making edits in adult cells, which are not passed down to offspring. We will not edit embryos, sperm cells or egg cells.
Safety is paramount at Verve. We will adhere to rigorous safety protocols with the best available technologies for detecting potential off-target effects. We have world-leading experts in human genetics, gene editing, LNP delivery and drug development on our team, and our clinical development plans will proceed responsibly.