GENE ONLINE|News &
Opinion
Blog

2022-07-19| Trials & Approvals

Verve Starts Human Trials for Gene Editing Medicine, Targeting Heterozygous Familial Hypercholesterolemia

by Nai Ye Yeat
Share To

Verve announced its novel gene-editing medicine VERVE-101, which is currently under Phase 1b clinical trial, having its very first patient getting dosed. VERVE-101 is targeting patients with heterozygous familial hypercholesterolemia (HeFH), a prevalent and life-threatening subtype of atherosclerotic cardiovascular disease (ASCVD). As an investigational gene-editing medicine, it was designed to be a single-course treatment that hoped to permanently turn off the PCSK9 gene in the liver to reduce low-density lipoprotein cholesterol (LDL-C), or known as “bad cholesterol”.

Related article: Landmark CRISPR Trial Shows Promise Against Fighting Blindness, but Is It a Cure?

 

CRISPR Technology in Disease Treatment

 

VERVE-101 consists of an adenine base editor messenger RNA and an optimized guide RNA targeting the PCSK9 gene packaged in an engineered lipid nanoparticle. It’s designed to change adenine in PCSK9 to guanine. The A-G swap will inactivate the PCSK9 gene, while the inactivation has been shown to up-regulate LDLR expression, which leads to lower LDL-C levels, hence reducing the risk for ASCVD.

The novel drug based on a more precise form of CRISPR known as base editing marked the latest milestone in CRISPR technology for altering human DNA to treat diseases.

 

The Ongoing Heart-1 Trial

 

“Preclinical data suggest that VERVE-101 has the potential to offer people with HeFH a game-changing treatment option, transforming the traditional chronic care model to a single-course, life-long treatment solution,” said Sekar Kathiresan, M.D., co-founder, and chief executive officer of Verve.

The current standard of care treatment for HeFH is highly dependent on patient adherence, lifestyles, health care access, and infrastructure, which largely limits the efficacy to achieve LDL-C goal levels. The low success rate of not more than 20% called for an urgent need for better treatment alternatives. VERVE-101 has the potential to break through the bottleneck of current treatment, and the company was optimistic to promote it to the millions of people with ASCVD globally.

The heart-1 clinical trial is planned to enroll approximately 40 adult HeFH patients with established ASCVD and the primary endpoint will focus on the safety and tolerability of VERVE-101 administration, with additional analyses for pharmacokinetics and blood PCSK9 protein and LDL-C reductions. The initial results are expected sometime in 2023, according to Verve.

©www.geneonline.com All rights reserved. Collaborate with us: service@geneonlineasia.com
Related Post
Arsenal Biosciences Announced Another $70 million Collaboration, This Time With Genentech
2022-09-27
AstraZeneca Blocks Ionis’ Hypercholesterolemia Treatment from Moving into a Phase 3 Study
2022-09-23
This One-Time Injection Could Cure AIDS
2022-06-21
LATEST
Junshi’s PD-1 Antibody Meets Endpoint In Phase 3 Lung Cancer Trial
2023-01-19
Moderna Welcomes Phase 3 Win For RSV Vaccine
2023-01-18
Moving Beyond COVID With mRNA Technology
2023-01-18
Rob Knight and Jing-Yuan Fu Elaborate On Microbiome Research Trends at the 7th Asia Microbiome Conference(AMC)
2023-01-17
CARsgen Taps Huadong to Commercialize Multiple Myeloma CAR-T in China
2023-01-17
Scientists Study Imaging Probes in First-Ever Amputated Human Limb Model
2023-01-16
EMA Plans to Issue Liver Failure Warning for Novartis’ Gene Therapy
2023-01-16
Scroll to Top

Create an account with us now to say goodbye to all the pop-ups!