Vertex axes Verve gene editing pact amid shift in R&D priorities

Vertex has backed out of its in vivo gene editing collaboration with Verve Therapeutics before reaching the clinic. Verve, which attributed Vertex’s action to its changing R&D priorities, is taking the program forward independently having regained full rights to the asset.

Vertex paid $25 million upfront and invested $35 million in Verve at the outset of the partnership in July 2022. The deal included up to $66 million in success payments and up to $340 million in development and commercial milestones. In return, Vertex secured the chance to collaborate with Verve for four years on the discovery and development of an in vivo gene editing program for a single liver disease.

Thursday, Verve told investors however that Vertex has now terminated the research collaboration “due to changing priorities within its development portfolio.” Verve has now regained rights to develop the preclinical program.

Vertex reimbursed the research up to the termination of the deal, covering the costs of Verve’s work to identify and engineer specific gene editing systems and in vivo delivery systems directed to the target and evaluate and optimize candidates. Under the terms of the now-defunct alliance, Vertex would have funded Verve through phase 1 and then decided whether to opt in and take over development.

The Vertex liver disease program is an outlier in a Verve pipeline focused on cardiovascular disease. At a Goldman Sachs event in June, Verve CEO Sekar Kathiresan, M.D., said the Vertex program is “the only product in our pipeline that is liver disease, and we chose to work with them because they have deep expertise in this disease.”

Vertex’s exit cuts Verve off from that expertise, but, having worked on a bespoke editor for the program, the biotech plans to take the candidate forward independently. Verve’s statement lacks a timeline for reaching the clinic.

The termination of the Vertex deal comes as Verve’s other big partnership nears a key moment. Verve is preparing to deliver the opt-in data package for its PCSK9 program to Eli Lilly in the second half of 2025. The biotech expects to receive a decision from Lilly this year. Initial results from a phase 1b trial of the in vivo, investigational base editing medicine are due in the second quarter.