Why Cardiovascular Disease
Despite advances in treatment over the last 50 years, cardiovascular disease (CVD) remains a global epidemic. The current paradigm of chronic care is fragile – requiring rigorous patient adherence, extensive healthcare infrastructure and regular healthcare access – and leaves many patients without adequate care. CVD is a leading contributor to reductions in life expectancy and is one of the most expensive health conditions in the U.S., costing the U.S. healthcare system more than $320 billion per year in annual costs and lost productivity.
CVD collectively refers to diseases of the heart and blood vessels, which are diagnosed as atherosclerotic cardiovascular disease (ASCVD) or cardiomyopathy, among others. In ASCVD, a large subset of CVD, cholesterol drives the development of atherosclerotic plaque, a mixture of cholesterol, cells and cellular debris in the wall of a blood vessel that results in the hardening of the arteries.
Limitations of Current Approaches
Only 39% of individuals reported adherence to statin therapy in the year following a heart attack, even when the medicine was provided free of charge.
Current therapies are given over decades with costs that can be prohibitively expensive.
Existing therapeutics carry side effects for a subset of the population.
The current treatment approach starts too late, after decades of exposure to damaging levels of risk factors.
Ongoing access to oral medications and specialist physicians is limited in developing countries where coronary heart disease is rising.
Only 39% of individuals reported adherence to statin therapy in the year following a heart attack, even when the medicine was provided free of charge.
Current therapies are given over decades with costs that can be prohibitively expensive.
Existing therapeutics carry side effects for a subset of the population.
The current treatment approach starts too late, after decades of exposure to damaging levels of risk factors.
Ongoing access to oral medications and specialist physicians is limited in developing countries where coronary heart disease is rising.
Genetics and Cardiovascular Disease
Human genetic studies have shown that certain individuals have gene variants that cause high LDL-C levels, leading to increased risk for ASCVD, including heart attack. Conversely, studies have found that certain gene variants confer natural disease resistance and reduce the risk for ASCVD. For instance, a subset of the population carries a naturally protective variant of a gene known as PCSK9. People born with this variant have extremely low LDL-C levels, are healthy and resistant to heart attack.
Our gene editing medicines are designed to mimic the protective effects of naturally occurring resistance mutations. By potently and durably lowering cumulative LDL-C exposure throughout a patient’s lifetime, our gene editing medicines could fundamentally disrupt the chronic care model for treating patients with or at risk for ASCVD and relieve the significant burden placed on patients, providers and the healthcare system.
Learn more about our approach