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Vertex, Verve team up to develop a gene editing drug for liver disease

Vertex Pharmaceuticals is broadening its reach in gene editing, announcing Wednesday a deal with Verve Therapeutics to develop a one-time treatment for an undisclosed liver disease.

Vertex will pay Verve $25 million in cash and make a $35 million investment in the smaller biotechnology company to kick off the four-year alliance. Verve could receive another $66 million in unspecified “success” payments, and $340 million more if the program hits certain development and commercial targets.

Vertex will fund early research conducted by Verve and lead clinical development of any program that comes from the work, the companies said.

The partnership is significant for Verve, a biotech co-founded by cardiologist and geneticist Sek Kathiresan that aims to develop a treatment that can prevent heart attacks for life. Verve raised $267 million in one of the larger biotech initial public offerings last year, and has since become the first company to test “base” editing — a more precise form of CRISPR gene editing — in a human clinical trial.

Verve shares closed Tuesday at just under $36 apiece, making it the top-performing biotech company to go public in 2021 and one of the few still trading above its debut price, according to data compiled by BioPharma Dive. (Shares fell by double digits in after-hours trading, though, as Verve simultaneously announced a $200 million stock offering.)

The deal expand’s Verve’s portfolio into a new area, liver diseases, while adding to Vertex’s growing ambitions in genetic medicine.

Best known for four successful cystic fibrosis medicines, Vertex has identified gene editing as a way to branch out its research. The company’s most advanced prospect is a treatment for sickle cell disease and beta thalassemia that’s being co-developed with CRISPR Therapeutics.

Vertex has invested in other areas in recent years, too, acquiring two biotechs making cell therapies for diabetes and another working on a gene editing treatment for Duchenne muscular dystrophy.

However, much of that work is focused on gene editing treatments that are built outside of the body. Verve’s drug candidates are designed to edit genes while inside the body, an approach also being pursued by Intellia Therapeutics and Editas Medicine, among a few others.

Vertex and Verve’s alliance builds on the long relationship between Kathiresan and Vertex R&D chief David Altshuler. Kathiresan worked for Altshuler at the Broad Institute of MIT and Harvard for nearly a decade while studying the genetic underpinnings of heart disease

“We are impressed by the progress Verve has made and look forward to combining the expertise in gene editing and drug development of our two companies to serve more patients in need,” Altshuler said in a statement.