Verve and Vertex Agree to Co-Develop In Vivo Gene Editing Program for Liver Disease

Verve Therapeutics and Vertex Pharmaceuticals are joining forces to develop an in vivo gene editing therapy for an undisclosed liver disease, the companies announced Wednesday. 

For the next four years, Verve will be responsible for the discovery and preclinical development of a candidate for the target of interest. Vertex will fund all program costs, and take over subsequent development, production and marketing of Verve’s candidate. 

Verve will receive a $25 million upfront payment, a $35 million equity investment, as well as up to $66 million in success payments and $340 million in milestones. The biotech will also reap tiered royalties on future net sales of any product resulting from this collaboration. 

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

The Potential of Base Editors 

After raising $267 million in an IPO last year, Verve became the first company to trial base editing in humans. Base editing, a form of gene editing, specifically changes a single letter in the patient’s DNA, giving new opportunities to fix disease-causing genes. The approach has the potential to be a more precise and safer alternative to CRISPR-Cas9 editing which may cause unintended changes after cutting DNA. 

The candidate Verve advanced into the clinic, VERVE-101, was co-developed with Beam Therapeutics and is designed to silence PCSK9 in the liver to treat heterozygous familial hypercholesterolemia, an inherited disease characterized by high levels of low-density lipoproteins (LDL) and an increased risk of heart attack. 

In an encouraging in vivo proof-of-concept study involving non-human primates, a single intravenous infusion of VERVE-101 resulted in 89% baseline reduction of PCSK9 protein in the blood, as well as 59% reduction in LDL at two weeks.  

Meanwhile, the company’s second gene editing program, targeting ANGPTL3 for familial hypercholesterolemia, is in the lead optimization phase. The Vertex collaboration will take Verve into a new direction — liver diseases. 

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Joy Lin

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