Genetic manipulation for elevated cholesterol alters body's DNA, lowering LDL levels.
### Revolutionary Gene Editing Therapy VERVE-102 Offers Hope for High Cholesterol Sufferers
A groundbreaking CRISPR-Cas9 gene editing therapy, VERVE-102, is making strides in the treatment of high cholesterol levels, particularly in conditions like familial hypercholesterolemia (FH) and the risk of premature coronary artery disease.
Recruitment for the fourth group of participants is currently underway in the United Kingdom, Canada, Israel, Australia, and New Zealand. The therapy targets the PCSK9 gene, which plays a crucial role in regulating low-density lipoprotein cholesterol (LDL-C). By inactivating PCSK9, VERVE-102 aims to increase LDL receptor expression, thereby lowering LDL-C levels.
The Food and Drug Administration (FDA) has granted VERVE-102 "Fast Track Designation" to expedite its development and approval. The experimental gene therapy, developed by Verve Therapeutics, uses a modification of the gene-editing system called base editing, which swaps out just one letter in DNA's code.
In the ongoing clinical trial, a single dose of VERVE-102 led to an average reduction of 53% in LDL levels. Four participants given the highest dose of VERVE-102 experienced a 69% reduction in LDL levels at maximum. The treatment is well-tolerated, with no treatment-related serious adverse events (SAEs) and no clinically significant laboratory abnormalities observed.
VERVE-102 is designed to reduce levels of low-density lipoprotein (LDL) cholesterol, often referred to as the "bad" kind of cholesterol. The new dosing regimen for VERVE-102 is a single intravenous infusion over two to four hours.
Ruth Gimeno, Lilly group vice president, believes VERVE-102 has the potential to shift the treatment paradigm for cardiovascular disease. The therapy targets PCSK9, especially in the liver, where LDL receptors are abundant.
As the trial progresses, Verve plans to release data from this portion later this year and start its next clinical trial, which will likely enroll U.S. participants. In June, Verve was acquired by the drug company Lilly, which aims to continue the development of the treatment.
Premature coronary artery disease (CAD), a condition in which the arteries narrow and can't deliver enough oxygenated blood to heart muscle, is generally considered to occur before age 65 in women and age 55 in men. By effectively lowering LDL-C, VERVE-102 could reduce the risk of premature CAD in individuals with FH.
VERVE-102 offers the potential for a one-time curative treatment, eliminating the need for lifelong statins or injectable PCSK9 inhibitors. This could significantly reduce the risk of atherosclerotic cardiovascular disease (ASCVD) associated with high cholesterol. However, gene editing therapies, including CRISPR, carry risks of off-target effects, efficient and safe delivery of gene editing components to the target site remains a challenge, and high regulatory hurdles exist for gene editing therapies, with no guarantee of FDA approval even if successful in trials.
Nevertheless, the promise of VERVE-102 as a revolutionary treatment for high cholesterol and related conditions continues to inspire hope for those affected by these conditions.
The revolutionary gene-editing therapy VERVE-102, targeting the PCSK9 gene, could potentially provide significant advancements in the health and wellness sector, particularly for individuals suffering from medical conditions like high cholesterol, familial hypercholesterolemia (FH), and the associated risk of premature coronary artery disease. This groundbreaking therapy, with its potential to reduce the need for lifelong treatments like statins, could revolutionize therapies and treatments for chronic diseases.
