Verve Doses First Patient with Investigational Gene-Editing Medicine

A recent statement by Verve Therapeutics announced the start of the phase 1b clinical trial on heterozygous familial hypercholesterolemia (HeFH) in New Zealand. This announcement highlighted the first administration of their investigational in vivo base gene-editing medicine called VERVE-101.  

HeFH is a subcategory of atherosclerotic cardiovascular disease (ASCVD). 

According to the CDC, familial hypercholesteremia affects approximately 1 in 250 people in the United States. Familial hypercholesteremia is characterized by an increased level of low-density lipoprotein cholesterol (LDL–C) in the blood.  

Mount Sinai Medical Center states that hypercholesterolemia increases the risk of heart disease, stroke, and insulin resistance. High LDL–C levels can double the risk of a heart attack.  

Traditional treatments for hypercholesterolemia include lifestyle changes, such as adopting a healthy diet and medications. Lifestyle changes may be beneficial but are often not sufficient treatment alone.  

Many patients can manage symptoms; however, medications may impact other conditions and cause side effects.  

“With the current standard of care treatment for HeFH, less than 20% of patients achieve LDL–C goal levels due to the limitations of the chronic model, which requires rigorous patient adherence, regular health care access, and extensive health care infrastructure. VERVE-101 has the potential to change the way cardiovascular disease is cared for by lowering LDL-C as low as possible for as long as possible after a single treatment,” stated Andrew Bellinger, MD, PhD, chief scientific and medical officer of Verve, in their press announcement.  

VERVE-101 functions by inhibiting the expression of the PCSK9 gene in the liver, lowering LDL–C. It is composed of adenine base editor messenger RNA (and an optimized guide RNA that target the appropriate gene and changes a single base in the genetic sequence to turn it off.  

The phase 1b clinical trial intends to enroll 40 adult HeFH patients with established ASCVD.  

“VERVE-101 is a first-in-class gene-editing medicine that we have designed to make a single spelling change in liver DNA to permanently turn off a disease-causing gene. The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing,” said Sekar Kathiresan, MD, co-founder and chief executive officer of Verve, in the release.  

The results of this clinical trial may be life-altering for patients and revolutionize care for this disease. Providers and patients alike wait in anticipation of clinical trial results.