Verve Therapeutics, Inc. is a genetic medicines company, which engages in the development of approach to the care of cardiovascular diseases. The company was founded by Burt A. Adelman, Keith J. Joung, Sekar Kathiresan, Kiran Musunuru, Anthony Philippakis, Issi Rozen, and Barry Ticho in 2018 and is headquartered in Boston, MA.
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成立时间
2018
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- Cellectis' board approved a strategic transformation on September 11, 2026, ending internal development of CAR-T candidates lasme-cel and eti-cel and refocusing on in vivo gene editing. - The company will advance two preclinical programs, .HEAL-101 targeting APOC3 for severe hypertriglyceridemia and .HEAL-201 targeting PCSK9 for severe hypercholesterolemia, both LNP-delivered. - Cellectis attributed the CAR-T exit to improved frontline regimens, bispecific antibody competition and slower enrollment, and will seek partners for the discontinued assets. - The operational realignment is designed to extend the cash runway into H2 2028, with preliminary Phase 1 data from .HEAL-101 expected in H2 2027 and .HEAL-201 in H1 2028.
- The ANGPTL3 inhibitors market was valued at USD 0.21 billion in 2025 and is projected to reach USD 3.48 billion by 2035, at a 32.4% CAGR. - Monoclonal antibodies led the market with 71% share in 2025, driven by Regeneron's EVKEEZA, the sole FDA-approved ANGPTL3 inhibitor for homozygous familial hypercholesterolemia. - Arrowhead's zodasiran is advancing into Phase III development following positive Phase IIb ARCHES-2 results, positioning RNAi therapeutics as the fastest-growing drug class. - Verve Therapeutics' VERV-201, an in vivo base-editing therapy, is advancing toward potentially one-time, permanent ANGPTL3 reduction for refractory hypercholesterolemia and HoFH.
- The novel hypercholesterolemia drugs market is projected to grow from USD 17.6 billion in 2026 to USD 42.8 billion by 2036, a 9.3% CAGR. - PCSK9 inhibitors are forecast to hold a 42.0% drug-class share in 2026, spanning monoclonal antibodies, inclisiran-based siRNA, and the new oral enlicitide. - Merck's Lipfendra (enlicitide) gained FDA approval in July 2026 as the first once-daily oral PCSK9 inhibitor for adults with hypercholesterolemia. - Injectable therapies lead with a 57.0% route-of-administration share, while gene-editing programs from Verve, CRISPR Therapeutics, and Eli Lilly advance toward one-time treatment.
- The global gene editing therapeutics market is projected to grow at a compound annual growth rate of 12.5%, fueled by expanding clinical applications and rising disease burden. - CRISPR-based technologies dominate the market due to their efficiency and cost-effectiveness, while oncology represents the largest therapeutic application segment. - High development costs, regulatory complexity, and off-target safety concerns remain significant barriers to broader adoption and commercialization. - Key players including CRISPR Therapeutics, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics are driving innovation through partnerships and investments in next-generation editing platforms.
- The FDA's Center for Biologics Evaluation and Research has released draft guidance establishing standardized methods for using next-generation sequencing to assess safety risks in genome editing therapies. - The guidance focuses on detecting off-target gene editing events and chromosomal translocations that could pose safety risks in both ex vivo and in vivo genome editing products. - FDA Commissioner Marty Makary emphasized that genome editing holds "extraordinary promise" for treating previously incurable genetic diseases and represents the agency's forward approach to advancing therapeutic development. - The framework complements previous January 2024 guidance and aims to support investigational new drug applications and biologics license applications for gene therapy products.
- Merck's oral non-statin drug enlicitide decanoate reduced bad cholesterol by up to 64.6% from baseline in an eight-week late-stage trial when added to background statin treatment. - The drug demonstrated superior efficacy compared to existing non-statin therapies, reducing LDL-C by 56.7% versus bempedoic acid and 36.0% versus ezetimibe in head-to-head comparisons. - Enlicitide works by blocking PCSK9 protein and could represent a significant pipeline asset for Merck as it seeks new blockbuster candidates ahead of Keytruda's patent expiration. - The drug has received FDA Commissioner's National Priority Voucher status, potentially enabling approval as early as 2026 for treating hypercholesterolemia affecting 73.5 million Americans.
- Eli Lilly has signed a strategic research and licensing agreement worth up to $1.1 billion with Dresden-based Seamless Therapeutics to develop gene therapies for genetically caused hearing loss. - The collaboration leverages Seamless's proprietary recombinase platform, which enables precise DNA insertions and modifications without relying on cellular DNA repair mechanisms, differentiating it from CRISPR/Cas technology. - The deal includes guaranteed upfront payments and committed R&D funding, with Lilly receiving exclusive licensing rights to advance candidates through preclinical and clinical development. - This partnership represents a significant validation of Seamless's gene editing platform and positions the company for potential additional collaborations while advancing their own internal programs.
- GenEditBio and ToolGen have entered a strategic cross-license agreement combining ToolGen's CRISPR-Cas9 platform with GenEditBio's lipid nanoparticle delivery technology. - The collaboration focuses on developing GEB-200, a once-and-done gene therapy targeting lipoprotein(a) for atherosclerotic cardiovascular disease affecting 20% of the global population. - The companies recently completed successful proof-of-concept studies in non-human primates demonstrating effective target gene modulation and favorable safety profiles. - The partnership aims to accelerate regulatory pathways and explore global licensing opportunities in the rapidly growing cardiovascular gene-editing market.
- Eli Lilly announced the acquisition of Adverum Biotechnologies for up to $261.7 million, including its lead gene therapy candidate Ixo-vec for wet age-related macular degeneration. - Ixo-vec is designed as a single intravitreal injection to provide continuous aflibercept levels, potentially replacing the current paradigm of repeated chronic anti-VEGF injections. - The gene therapy is currently being evaluated in the Phase 3 ARTEMIS trial and has received Fast Track and RMAT designations from the FDA. - This acquisition represents Lilly's second major gene therapy investment in 2025, following the $1.3 billion Verve Therapeutics deal announced in June.
- Eli Lilly will acquire gene-editing startup Verve Therapeutics for up to $1.3 billion, including an upfront payment of almost $1 billion and $300 million in milestone-based payments. - The acquisition strengthens Lilly's pipeline beyond its blockbuster weight-loss and diabetes drugs, focusing on one-time gene-editing therapies for high cholesterol in heart disease patients. - Verve's lead therapies use base editing technology to target PCSK9, ANGPTL3, and LPA genes responsible for regulating blood cholesterol levels. - The companies were already partnering on cholesterol-lowering treatments, with Verve's VERVE-102 therapy currently in early-stage trials for familial hypercholesterolemia.
