Eli Lilly is pushing a gene-editing therapy into Phase 2 that could permanently slash cholesterol with a single infusion. It's the biggest bet yet that CRISPR-era medicine can tackle the world's leading killer, not just rare diseases.
The Pill You'll Never Have to Take Again
Imagine a world where your doctor doesn't prescribe you a daily statin. Instead, you get a single IV infusion, walk out of the clinic, and your "bad" cholesterol drops by more than half. Permanently. No refills. No forgetting your evening pill. No twice-yearly injections.
That world just got a lot closer. Eli Lilly is planning to advance VERVE-102, a gene-editing therapy for high cholesterol, into Phase 2 clinical trials in the second half of 2025. The drug works by rewriting a single letter of DNA in your liver cells, flipping off the gene responsible for keeping your LDL cholesterol high. One treatment. Done.
For a company best known for diabetes and obesity drugs, this is a massive bet on the future of cardiovascular medicine. And Wall Street is paying attention.
How You Edit a Gene to Lower Cholesterol
To understand why this matters, you need to know about a protein called PCSK9. Think of PCSK9 as a bouncer at a nightclub. Its job is to escort LDL receptors (the things that pull bad cholesterol out of your blood) off the surface of your liver cells and destroy them. More PCSK9 means fewer receptors, which means more cholesterol floating around clogging your arteries.
Drugs that block PCSK9 already exist. Repatha and Praluent are injectable antibodies that tackle the protein head-on. Novartis sells Leqvio (inclisiran), an RNA-based drug that silences PCSK9 production twice a year. They all work reasonably well, cutting LDL by roughly 50 to 60 percent.
But they all share the same problem: you have to keep taking them. Stop the drug, and your cholesterol bounces right back.
VERVE-102 takes a fundamentally different approach. Developed by Verve Therapeutics (which Lilly acquired for about $1.3 billion in 2025), the therapy uses a technology called adenine base editing. It's a cousin of CRISPR, but more precise. Instead of cutting DNA like molecular scissors, it chemically converts a single A to a G in the PCSK9 gene. No double-strand breaks. No dramatic genomic surgery. Just a quiet, permanent typo that renders PCSK9 useless.
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The edit gets delivered to liver cells via lipid nanoparticles (tiny fat bubbles, essentially the same delivery tech that powered the COVID mRNA vaccines). One infusion, and the bouncer is fired for good.
The Numbers That Got Lilly Excited
In Phase 1b testing, VERVE-102 delivered results that made analysts sit up in their chairs. At the highest dose tested (0.6 mg/kg), patients saw a mean LDL cholesterol drop of 53 percent after a single infusion, with a maximum individual reduction of 69 percent. PCSK9 protein levels fell by a mean of 60 percent, and early data suggested those effects held steady for up to 18 months.
No serious treatment-related safety events were reported in the initial cohort of 14 patients with at least 28 days of follow-up. William Blair analyst Myles Minter called the data a "clear win" that appeared to resolve safety concerns from Verve's earlier program. Jefferies analyst Roger Song went further, arguing VERVE-102 shows "best-in-class potential" versus existing PCSK9 therapies, including Novartis's Leqvio.
Those are strong words for a drug this early. But the numbers back them up: comparable LDL reduction to injected antibodies, better than inclisiran, and you only have to do it once.
Why Lilly Spent $1.3 Billion on a Startup
Lilly didn't stumble into this acquisition. The company had been circling Verve for years, building its position like a chess player setting up a checkmate.
In 2023, Lilly paid Verve $60 million upfront for a preclinical cardiovascular gene-editing partnership. Around the same time, it paid Beam Therapeutics $250 million for opt-in rights to Verve's key programs (Beam had licensed its base-editing technology to Verve and held economic rights). By mid-2025, Lilly decided to stop renting and buy the whole house: $10.50 per share in cash (roughly $1 billion upfront), plus up to $3.00 per share in milestone payments tied to starting a U.S. Phase 3 trial.
The acquisition gave Lilly full control over three gene-editing programs that systematically attack the major drivers of heart disease. VERVE-102 targets PCSK9 for LDL cholesterol. VERVE-201 targets ANGPTL3, a protein involved in triglycerides and treatment-resistant cholesterol. VERVE-301 goes after lipoprotein(a), a genetically determined risk factor for heart attacks and strokes that has almost no approved treatments.
Pair those with Lilly's existing obesity and diabetes franchise, and you can see the strategic vision: a company that can address weight, blood sugar, and lifetime cardiovascular risk, all under one roof.
The One-and-Done Dream (and Its Skeptics)
The idea of editing away heart disease risk with a single clinic visit sounds almost too good to be true. And some healthy skepticism is warranted.
For starters, this is a permanent change to your DNA. That's the whole selling point, but it's also the biggest risk. If something goes wrong (unexpected off-target edits, long-term liver effects, immune reactions to the editing machinery), you can't un-ring that bell. Regulators at the FDA will want years of follow-up data before they're comfortable approving a gene edit for a condition that already has safe, reversible treatments on the market.
Then there's the adoption question. Cardiologists are used to prescribing pills they can stop if side effects emerge. Asking them to recommend a permanent genetic alteration requires a fundamentally different risk-benefit conversation with patients. It's the difference between renting an apartment and buying a house with no return policy.
Pricing will be another puzzle. Gene therapies for rare diseases can cost $1 to $3 million per patient because the patient populations are tiny. Cholesterol affects hundreds of millions of people worldwide. A gene-therapy price tag doesn't scale to that kind of population, but a conventional drug price doesn't reflect the value of a one-time cure. Expect creative payment models: installment plans, outcomes-based contracts, maybe something entirely new.
A Bigger Story Than One Drug
Lilly's planned Phase 2 advancement matters beyond the LDL numbers. It marks a turning point for gene editing as a technology platform.
Until recently, in vivo gene editing (editing genes inside a living person, rather than removing cells, editing them, and putting them back) was confined to ultra-rare diseases. Intellia's NTLA-2001 for a rare heart condition called ATTR amyloidosis was the proof of concept. But cardiovascular disease kills more people than any other cause on Earth. Moving gene editing into this space is like taking a sports car off the test track and onto the highway.
Lilly isn't alone, either. CRISPR Therapeutics has an in vivo program (CTX310) targeting ANGPTL3 that showed roughly 50% LDL and 55% triglyceride reductions in Phase 1, with Phase 1/1b data updates expected in the second half of 2026. Novo Nordisk has partnered with Life Edit on cardiometabolic gene-editing therapies. Scribe Therapeutics reported its editing platform lowered LDL by up to 67% in primates.
The race is on. And the fact that multiple large pharma companies are now writing billion-dollar checks tells you something important: the industry believes one-shot cardiovascular gene editing isn't science fiction anymore. It's a pipeline strategy.
For patients currently juggling statins, PCSK9 injections, and fish oil capsules, that future can't arrive soon enough.
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