Verve snags $60M from Lilly for one-and-done cardio gene editing

Eli Lilly has tapped another gene editing partner, this time looking to go after a large patient population in the cardiovascular space with the help of Verve Therapeutics.

As part of an in vivo gene editing deal, the Indianapolis Big Pharma will pay the Boston biotech $30 million in cash, invest $30 million via equity at a 15% premium to the 30-day volume-weighted average price, and contribute up to another $465 million in R&D and commercial milestones, Verve CEO and co-founder Sekar Kathiresan told Endpoints News.

Sek Kathiresan

On top of that, Lilly will pay for research through a Verve-led Phase I clinical trial, after which Verve can opt to co-fund and share margins globally in lieu of the milestones and tiered royalties, the companies said Thursday morning. Lilly’s investment is expected to help keep Verve’s lights on into 2026.

The program focuses on Lipoprotein (a), or Lp(a), which Kathiresan described as LDL’s cousin. The particle carries cholesterol and linked to one of the surface proteins is apolipoprotein(a). Lp(a) is produced in the liver, travels throughout the blood and can promote blood clotting. About 11 million Americans have elevated levels of the particle, the CEO added, and it’s a big risk factor in heart, stroke and other diseases. Nearly one in five people in the US and Europe who have heart disease have elevated Lp(a), he said.

The program is still nascent — the company just announced the target in January, and it’s part of the biotech’s broader mission to tackle atherosclerotic cardiovascular disease with the latest gene editing approaches.

The deal adds to Lilly’s deep history in the cardiometabolic space across diabetes and obesity. It also marks another gene editing collaboration for the pharma, whose Prevail unit disclosed a Scribe Therapeutics tie-up last month.

Verve hopes to create one-and-done treatments, whereas Big Pharmas like Novartis and Amgen are making injectables that need to be administered to patients multiple times.

By the time Verve enters the clinic for elevated levels of Lp(A), two treatments could be nearing market entry; Novartis’ pelacarsen is expected to have Phase III topline data in 2025, and Amgen’s siRNA olpasiran is set to have late-stage data in 2026. Novartis has said it sees multibillion-dollar potential in its investigational med, in-licensed from Ionis.

Those therapies will “set the stage, hopefully,” for Verve’s gene editing approach, Kathiresan said.

“We really think the chronic care model still leads to a heavy treatment burden,” he added.

Verve plans to start Phase I testing during the five-year collaboration with Lilly, which is pending regulatory clearance. Unlike its first clinical-stage program, which targets PCSK9, Verve will not use a Beam Therapeutics-allied base editor for the Lp(a) program. Instead, Verve is building a custom editor, Kathiresan said.

Based on research during his time at Massachusetts General Hospital and Harvard Medical School, Kathiresan and his lab made observations about gene mutations that turn off Lp(a) and bring their levels down, which can help protect against heart diseases.

In the gene that Verve wants to turn off, there are several thousand letters that can go haywire and get repeated once or dozens of times. The so-called kringle IV repeats (determined by the DNA inherited) make the editing process “potentially a little more complicated,” Kathiresan said.

Another reason to make a custom editor is that the targeted gene is similar to other genes, like plasminogen, Kathiresan said.

Lilly had approached the company a year ago, prior to Verve’s disclosure of a Vertex deal around a type of liver disease, according to Kathiresan. That means Verve now has a “pretty full plate” with five targets and six programs (two PCSK9-directed) that are all in vivo in the liver.

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