Eli Lilly and Company said on May 25, 2026, that its experimental gene-editing medicine Verve 102 produced durable reductions in LDL cholesterol in a phase 1b study, with effects lasting as long as 18 months after a single infusion. The company said the treatment was well tolerated, with no treatment-related serious adverse events or dose-limiting toxicities reported.
The findings came from the Heart-2 trial, which is testing Verve 102 in adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. In the interim analysis of 35 participants, the drug lowered PCSK9 by a mean 51% to 88% across doses from 0.3 mg/kg to 1.0 mg/kg, while LDL-C fell by a mean 9% to 62% across dose levels. All participants received the full planned dose, and no one withdrew from the study.
The data were presented as a late-breaking oral presentation at the European Atherosclerosis Society Congress and published at the same time in The New England Journal of Medicine, putting the results in front of both clinicians and investors on the same day. Lilly said it plans to begin enrolling the phase 2 study of Verve 102 by the end of 2026, a step that will matter because the drug is being positioned as a one-time approach to a problem that usually requires lifelong treatment.
Verve 102 is an investigational in vivo base editing medicine designed to durably turn off the PCSK9 gene in the liver and lower blood LDL-C after a single infusion. The target matters because naturally occurring loss-of-function variants in PCSK9 are associated with markedly lower lifetime risk of coronary heart disease, and the U.S. Food and Drug Administration has already granted the drug Fast Track designation.
That is the promise driving the program, and also the pressure on the next phase. The early signal is strong, but phase 1b data are still small and built to test safety and dosing, not prove long-term clinical benefit. For Lilly, the question now is whether the response holds as more patients are enrolled and whether the treatment can keep its tolerability profile as the program moves toward a larger trial before the end of 2026.
