Verve is latest gene editing company to suffer setback, as Vertex walks away from pact

Verve Therapeutics has not escaped the doom that seems to be engulfing the gene editing sector of late. The company said Thursday that Vertex Pharmaceuticals handed back the rights to a preclinical-stage gene editing program for liver disease.

The two companies had agreed to a four-year collaboration in 2022 to investigate in vivo gene editing candidates for the treatment of a single liver disease, though both the specific disease and the genetic target were undisclosed. Vertex paid $25 million upfront and made a $35 million equity investment in Verve. Vertex also paid for the research, and Verve received collaboration revenue of $11.9 million under the agreement in 2024, according to an SEC filing.

Vertex’s decision to walk away from the pact, “due to changing priorities within its development portfolio,” adds more pressure to Verve’s other gene editing candidates.

Chief among these is VERVE-102, a program designed to permanently turn off the PCSK9 gene in the liver, thereby reducing cholesterol. It is in Phase 1b with Heart-2, an open-label study in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease, with initial data due next quarter.

Verve said that VERVE-102 has been well-tolerated so far, with the trial finding no treatment-related serious side effects or clinically significant laboratory abnormalities. Verve last year had to pause dosing in a Phase 1b trial of an earlier PCSK9-targeting gene editor, VERVE-101, after one patient had grade 3 elevation of the liver enzyme ALT and thrombocytopenia.

Final data from the dose escalation portion of the Heart-2 trial are expected in the second half of 2025. Analysts from William Blair wrote in a Feb. 21 note that they believe data from this trial could match the LDL-C reductions of 40% to 50% that were seen in trials of Novartis’ anti-PCSK9 RNA therapy Leqvio.

Under a different partnership deal Verve signed in 2023, Lilly has the right to opt in to VERVE-102 after Phase 1 studies conclude. Verve said it would get Lilly’s decision in the second half of this year.

The Lilly agreement also covers VERVE-101, which is still in limbo, and a program called VERVE-201 which targets ANGPTL3, with the aim of treating atherosclerotic cardiovascular disease. Verve has a separate deal with Lilly for a research-stage project, VERVE-301, which aims to reduce levels of lipoprotein (a).

Verve’s investors can at least be glad the company’s situation is not as bad as some others in the gene editing sector. CRISPR delivery startup Spotlight Therapeutics recently shut down, and Tome Biosciences and SalioGen Therapeutics met the same fate last year.

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