Gene editing specialist Verve Therapeutics has drawn a large pharmaceutical partner to its hunt for one-time treatments for heart disease, announcing Thursday a five-year research partnership with Eli Lilly.
Lilly will pay Verve, a buzzy biotechnology company that went public in 2021, $60 million in cash and equity to work together on a medicine that targets a cardiovascular risk factor known as lipoprotein(a), or Lp(a). Verve will be responsible for advancing a therapy into and through initial clinical testing in humans, after which Lilly would take over.
“Their attraction to us and our attraction to them is the shared interest in Lp(a),” said Verve CEO Sek Kathiresan, who described the liver-produced protein particle as a third “pillar” of artery-clogging risk alongside LDL cholesterol and triglycerides.
Prior studies have linked elevated Lp(a) levels with higher risk of heart disease, stroke and blood clots. But unlike with LDL cholesterol or triglycerides, no treatments exist to specifically reduce Lp(a) levels, which are largely determined by a person’s genetics.
That could change in the near future, as both Novartis and Amgen are studying Lp(a)-lowering injections in large clinical trials meant to prove the drugs can reduce heart risk. Results are expected in 2025 and 2026, respectively. Lilly is also testing a similar kind of medicine that it licensed from Dicerna Pharmaceuticals, and now has in a Phase 2 trial.
Verve is taking a different approach, aiming to tamp down Lp(a) by editing genes. Its calling card is a precise form of DNA modification known as base editing, technology for which it licensed from Beam Therapeutics. However, for the Lp(a) research program, Verve plans to use a custom editor it developed in house, Kathiresan said.
Rather than regular shots to keep Lp(a) levels under control, Verve instead envisions a one-time treatment that could result in permanent lowering — similar to how more advanced medicines it’s developing might keep LDL cholesterol in check for many years.
Lilly was interested in Verve’s plans to apply gene editing to widely prevalent diseases like heart disease, and approached the company about a year ago, according to Kathiresan.
“We see them as a great long-term partner,” he added, “because not only of their expertise in cardiovascular disease but also in commercialization of medicines for large indications.”
The deal is another dose of validation for Verve, which Kathiresan co-founded after a career as a cardiologist and geneticist.
The company was the first to test a base editing treatment in humans, although that study is on hold in the U.S. after regulators asked for more information. Last year, the company also inked a partnership with Vertex Pharmaceuticals to apply its technology in liver disease.
The Lilly deal is similar in scope to the one with Vertex, providing Verve with the same amount upfront but running one year longer.
The additional $60 million will give Kathiresan’s company enough cash to fund its operations into 2026, a two-and-a-half-year “budget envelope” that the CEO said could allow it to obtain clinical data for three different programs.