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28 HEALTH Friday, 24 November 2023
Groundbreaking CRISPR Treatment Shows Promise
in Reducing Cholesterol Levels in First Human Trial
A significant leap forward, CRISPR-based therapies have throughout the body.
achieved a breakthrough in a small clinical trial focused
on individuals genetically predisposed to dangerously high Despite the promising results, including a notable drop in
cholesterol levels. Led by Verve Therapeutics, the trial PCSK9 protein levels and circulating LDL, there were safety
involved ten participants, and a single infusion of the preci- considerations. Two participants experienced severe heart
sion gene editor successfully reduced artery-clogging fat by issues, prompting further evaluation. The treatment’s safety
up to 55 percent. was deemed acceptable by an independent monitoring
The innovative treatment, known as VERVE-101, employs board, but uncertainties remain, including potential off-
base editing technology—a more precise and potentially target effects and the long-term safety and efficacy of the
safer class of gene editors—making it the first of its kind to therapy.
be tested directly in humans for a chronic disease.
Dr. Sekar Kathiresan, CEO and cofounder of Verve, expressed
Unlike traditional approaches that involve daily medication, excitement about the positive initial data, emphasizing the
this one-shot CRISPR treatment, if successful, could offer significant potential for in vivo liver gene editing to treat
a lifetime solution for managing high cholesterol levels. familial hypercholesterolemia. The therapy has received
The trial primarily aimed to assess safety, and while not approval for testing in the United States, with plans to enroll
all participants responded positively, the results indicate a approximately 40 additional patients.
significant step forward in the potential treatment of chronic
conditions. Looking ahead, Verve is developing VERVE-102, a new
version of the therapy that incorporates similar base editing
The targeted gene in this trial was PCSK9, a liver protein technology and an upgraded nanoparticle carrier. If subse-
regulating “bad cholesterol” levels. Verve’s treatment quent trials prove successful, the company aims to initiate a
utilized two key strategies to enhance its effectiveness. First, randomized, placebo-controlled trial by 2025. While the cost
base editing, a more precise CRISPR tool, was employed to of the therapy remains undisclosed, the potential impact
disable the gene encoding the problematic liver protein. extends beyond familial hypercholesterolemia, offering a
Second, the treatment was delivered through nanoparticles, promising avenue for addressing high cholesterol as a prev-
addressing concerns about potential widespread editing alent health concern and mitigating the risk of heart disease.
