Eli Lilly's Verve-102 Gene Therapy Shows Promise for High Cholesterol
Eli Lilly announced on May 26, 2026, that the FDA granted fast-track designation to its VERVE-102 gene therapy for high cholesterol, following positive Phase 1b results. As of May 26, 2026, Eli Lilly plans to initiate Phase 2 studies by the end of 2026. The Phase 1b Heart-2 study, which began dosing patients on February 15, 2024, showed VERVE-102 successfully reduced PCSK9 protein levels by up to 88% and LDL cholesterol by as much as 62% in patients, with reductions sustained for at least 18 months and no serious adverse events reported. Eli Lilly acquired Verve Therapeutics, including VERVE-102, on June 15, 2025, to bolster its cardiovascular disease portfolio.
Timeline
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May 2026 — 2 developments
Eli Lilly announced that the FDA has granted fast-track designation to its VERVE-102 gene therapy for high cholesterol.
Eli Lilly announced that the FDA has granted fast-track designation to its VERVE-102 gene therapy for high cholesterol. The company plans to initiate Phase 2 studies by the end of 2026, following promising Phase 1b results showing significant and durable reductions in PCSK9 protein and LDL cholesterol levels.
Eli Lilly announced positive early results from the Phase 1b Heart-2 study of its experimental gene-editing therapy, Verve-102.
Eli Lilly announced positive early results from the Phase 1b Heart-2 study of its experimental gene-editing therapy, Verve-102. The therapy successfully reduced PCSK9 protein levels by up to 88% and LDL cholesterol by as much as 62% in patients with high cholesterol. These significant reductions were sustained for at least 18 months, with no serious adverse events reported, demonstrating strong potential for a one-time treatment.
March 2026 — 1 developments
Eli Lilly began preparations to publicly announce significant interim results from the VERVE-102 Phase 1b Heart-2 study.
Eli Lilly began preparations to publicly announce significant interim results from the VERVE-102 Phase 1b Heart-2 study. The company compiled and analyzed the comprehensive data, indicating strong efficacy and a favorable safety profile. This internal readiness signaled an upcoming major update on the gene therapy's progress, generating anticipation within the medical and investment communities.
September 2025 — 1 developments
Following the acquisition, Eli Lilly confirmed its commitment to the ongoing VERVE-102 Phase 1b Heart-2 study, continuing patient enrollment and data collection.
Following the acquisition, Eli Lilly confirmed its commitment to the ongoing VERVE-102 Phase 1b Heart-2 study, continuing patient enrollment and data collection. Lilly's resources and expertise were leveraged to potentially expand the study's scope or accelerate its progress. The seamless transition ensured the uninterrupted development of this promising gene therapy candidate.
June 2025 — 1 developments
Eli Lilly and Company announced its acquisition of Verve Therapeutics, a significant move that broug…
Eli Lilly and Company announced its acquisition of Verve Therapeutics, a significant move that brought Verve's innovative gene-editing pipeline, including VERVE-102, under Lilly's umbrella. This acquisition, valued at a substantial sum, aimed to bolster Eli Lilly's cardiovascular disease portfolio with a potential one-time treatment for high cholesterol. The integration was expected to accelerate the development and commercialization of VERVE-102.
January 2025 — 1 developments
As the Phase 1b Heart-2 study progressed, interim efficacy data for VERVE-102 began to show promising trends in reducing PCSK9 protein and LDL cholesterol levels.
As the Phase 1b Heart-2 study progressed, interim efficacy data for VERVE-102 began to show promising trends in reducing PCSK9 protein and LDL cholesterol levels. While not yet conclusive, these early signals suggested that the therapy was achieving its intended biological effect. The data reinforced the potential of VERVE-102 as a one-time treatment for high cholesterol.
July 2024 — 1 developments
Verve Therapeutics provided an early update on the Phase 1b Heart-2 study for VERVE-102, reporting initial positive safety data from the first cohorts of patients.
Verve Therapeutics provided an early update on the Phase 1b Heart-2 study for VERVE-102, reporting initial positive safety data from the first cohorts of patients. The preliminary findings indicated a favorable safety profile, with no serious adverse events directly attributed to the therapy. These early results were crucial for building confidence in the optimized delivery system and the overall therapeutic approach.
February 2024 — 1 developments
Verve Therapeutics announced the dosing of the first patient in its Phase 1b Heart-2 clinical study for VERVE-102.
Verve Therapeutics announced the dosing of the first patient in its Phase 1b Heart-2 clinical study for VERVE-102. This marked the official start of human trials for the company's optimized in vivo base-editing therapy, designed to permanently inactivate the PCSK9 gene. The study aimed to assess the safety, tolerability, and preliminary efficacy of VERVE-102 in patients with elevated LDL cholesterol.
March 2023 — 1 developments
Verve Therapeutics presented new preclinical data for VERVE-102, highlighting the improvements made to its delivery system compared to VERVE-101.
Verve Therapeutics presented new preclinical data for VERVE-102, highlighting the improvements made to its delivery system compared to VERVE-101. The data demonstrated that the optimized lipid nanoparticle (LNP) in VERVE-102 maintained potent PCSK9 inactivation and LDL cholesterol reduction in animal models, with an enhanced safety profile. These results supported the decision to advance VERVE-102 into clinical development.
October 2022 — 1 developments
In response to the safety concerns identified with VERVE-101's delivery system, Verve Therapeutics initiated the development of VERVE-102.
In response to the safety concerns identified with VERVE-101's delivery system, Verve Therapeutics initiated the development of VERVE-102. This new candidate was designed as a direct successor, incorporating an optimized lipid nanoparticle (LNP) delivery system while retaining the same base-editing mechanism targeting the PCSK9 gene. The goal was to improve the safety profile while maintaining high efficacy.
July 2022 — 1 developments
During the ongoing Phase 1 clinical trial for VERVE-101, early safety signals began to emerge, specifically related to the therapy's lipid nanoparticle (LNP) delivery system.
During the ongoing Phase 1 clinical trial for VERVE-101, early safety signals began to emerge, specifically related to the therapy's lipid nanoparticle (LNP) delivery system. While the gene-editing mechanism itself showed promise, concerns arose regarding the tolerability or potential off-target effects associated with the LNP. These observations prompted Verve Therapeutics to re-evaluate the delivery platform.
November 2021 — 1 developments
Verve Therapeutics announced that its Investigational New Drug (IND) application for VERVE-101 had b…
Verve Therapeutics announced that its Investigational New Drug (IND) application for VERVE-101 had been cleared by regulatory bodies, allowing the company to proceed with human clinical trials. This clearance was a significant milestone, paving the way for the first in-human studies of their innovative gene-editing therapy designed to lower LDL cholesterol. The company began preparations for patient enrollment.
August 2021 — 1 developments
Following encouraging preclinical results, Verve Therapeutics submitted an Investigational New Drug (IND) application to regulatory authorities for VERVE-101.
Following encouraging preclinical results, Verve Therapeutics submitted an Investigational New Drug (IND) application to regulatory authorities for VERVE-101. This filing marked a critical step towards initiating human clinical trials for their lead gene-editing candidate. The IND package included comprehensive data on the therapy's safety, manufacturing, and proposed clinical study design.
May 2021 — 1 developments
Verve Therapeutics presented promising preclinical data for its lead gene-editing candidate, VERVE-1…
Verve Therapeutics presented promising preclinical data for its lead gene-editing candidate, VERVE-101, at the American Society of Gene and Cell Therapy (ASGCT) 24th Virtual Annual Meeting. The data demonstrated that a single dose of the PCSK9-targeted gene editor led to potent and durable reductions of approximately 90% in blood PCSK9 protein and 60% in LDL-C levels in non-human primates. These results supported advancing VERVE-101 towards clinical development.
January 2021 — 1 developments
Verve Therapeutics secured a substantial $94 million in Series B funding, co-led by Wellington Management and Casdin Capital.
Verve Therapeutics secured a substantial $94 million in Series B funding, co-led by Wellington Management and Casdin Capital. This significant investment provided the company with ample resources to accelerate the development of its lead gene-editing programs, which included candidates targeting PCSK9 and ANGPTL3. The funding was a critical step as Verve prepared for its transition to the public market.
June 2020 — 1 developments
Verve Therapeutics announced the successful completion of a $63 million Series A2 funding round, with Alphabet's GV unit once again leading the investment.
Verve Therapeutics announced the successful completion of a $63 million Series A2 funding round, with Alphabet's GV unit once again leading the investment. This additional capital infusion was vital for further advancing the company's preclinical programs and expanding its research into gene-editing therapies. The continued investor confidence underscored the growing potential of Verve's innovative approach to addressing cardiovascular disease.
June 2019 — 1 developments
Verve Therapeutics launched its operations and successfully closed a $58.
Verve Therapeutics launched its operations and successfully closed a $58.5 million Series A funding round. This crucial initial investment was led by Alphabet's GV unit, with additional contributions from prominent investors such as Fidelity's F-Prime Capital, Biomatics Capital, and Arch Venture Partners. The funding was earmarked to accelerate the company's foundational research and development efforts in gene editing, particularly for cardiovascular diseases.
March 2018 — 1 developments
Verve Therapeutics was officially incorporated with a clear mission to pioneer single-course gene-editing medicines for cardiovascular conditions.
Verve Therapeutics was officially incorporated with a clear mission to pioneer single-course gene-editing medicines for cardiovascular conditions. The company was established by a team of experts in cardiology, gene editing, and drug development, including Sekar Kathiresan, Kiran Musunuru, and J. Keith Joung. Their foundational work focused on leveraging insights from human genetic analysis to develop innovative gene-editing technologies aimed at transforming the treatment landscape for coronary artery disease.