- Verve Therapeutics, a gene editing company vying to develop a one-time treatment for cardiovascular disease, has received regulatory clearance to begin its first study in humans.
- The Phase 1 trial will begin in New Zealand in mid-2022 and should produce initial data in 2023, Verve said in a statement. The company also plans to conduct clinical studies in the U.K. and U.S. and said it will file applications with regulators seeking permission for that research in the second half of this year.
- The company’s VERVE-101 treatment is designed to permanently turn off a gene called PCSK9 that plays an important role in controlling cholesterol levels in the blood. In the first clinical trial, the company will test the therapy in patients with a rare, genetic form of heart disease known as heterozygous familial hypercholesterolemia.
New Zealand’s decision to clear human testing represents a notable achievement for Verve and another important milestone for the type of gene editing technology the company is developing. Known as base editing, it's a potentially more precise way to modify genes, enabling scientists and drugmakers to change single DNA "letters."
Base editing builds on the progress made with CRISPR-based editing and has rapidly moved from laboratories into clinics. Beam Therapeutics, which licenses technology to Verve, won U.S. approval in November to begin a clinical trial of a base editing therapy for sickle cell disease. Unlike Beam's treatment, Verve's is designed to do its work inside the body, rather than on cells taken out of the body and modified in a lab. It's now the third inside-the-body gene editing treatment to enter human trials, following experimental drugs from Intellia Therapeutics and Editas Medicine.
While Tuesday's news is a milestone, Verve has a long way to go. Though the company is initially targeting a rare condition, it ultimately seeks to conquer one of the most prevalent diseases in the world. Even if the technology succeeds, Verve will have to persuade doctors and patients to adopt a potentially expensive genetic therapy when other, comparatively cheaper drugs exist to lower cholesterol and prevent some of the worst effects of heart disease.
For example, drugmakers in recent years have introduced several injectable medicines that specifically target PCSK9. While they had dramatic effects on cholesterol levels, insurers balked at the initial prices. The drugs are mostly used for patients at the most risk of a heart attack.
Verve CEO Sekar Kathiresan argues that his company's approach, if proven in clinical trials, offers an approach that will not only save lives, but also money for the healthcare system. He envisions a therapy akin to a vaccine for heart attacks that’s not dependent on taking pills or receiving injections for life.
Verve has been publicly traded for less than a year, having raised $267 million in an initial public offering in June. While its IPO was one of the most successful in the biotech industry in 2021, the company’s share price has tumbled along with its biotech peers in recent months. Once soaring above $70, Verve shares now trade around $13 apiece.