In a groundbreaking convergence of biotech innovation and cardiovascular medicine, Dr. Sekar Kathiresan, renowned cardiologist and geneticist, is steering his gene-editing company Verve Therapeutics toward a landmark $1.3 billion deal with Eli Lilly. If finalized, the agreement could fast-track the first in vivo gene-editing treatment aimed at dramatically reducing cholesterol—and with it, the global burden of atherosclerotic cardiovascular disease (ASCVD).
The prospective deal isn’t just about licensing. It represents a strategic alliance at the frontier of preventive medicine, where a single shot could permanently switch off genes responsible for high cholesterol, transforming how we treat and prevent heart disease in the coming decades.
And at the center of it all is Dr. Kathiresan—a physician-scientist who has made it his life’s mission to eliminate heart attacks from human history.
The Vision: A One-Time Genetic Fix for High Cholesterol
Verve Therapeutics, co-founded by Dr. Kathiresan in 2018, focuses on precise, one-time gene editing therapies delivered directly into the body. The company’s lead candidate, VERVE-101, targets the PCSK9 gene, which plays a key role in controlling LDL cholesterol—the “bad cholesterol” that clogs arteries and leads to heart attacks.
By delivering a base-editing payload via lipid nanoparticles (similar to mRNA vaccines), VERVE-101 permanently disables the PCSK9 gene in liver cells. The result? Lifetime reductions in LDL levels by over 60% after a single treatment.
Unlike statins or injectables like Repatha or Leqvio, which require ongoing dosing, Verve’s approach offers a “fire-and-forget” solution, with trials showing durable results well past 12 months.
The Deal: Lilly Bets Big on Genetic Prevention
Eli Lilly’s proposed $1.3 billion deal with Verve includes:
- $300 million upfront payment
- Up to $1 billion in milestone payments based on regulatory and commercial achievements
- Global co-development and co-commercialization rights to VERVE-101 and next-generation programs
- Joint research into new in vivo base-editing therapies targeting additional cardiovascular risk genes like ANGPTL3 and APOB
This partnership positions Lilly, already a diabetes and obesity pharma giant, as a serious contender in the genetic medicine space, competing with rivals like Novartis, Amgen, and CRISPR Therapeutics.
Verve, meanwhile, gains access to Lilly’s global manufacturing, trial infrastructure, and regulatory muscle, critical for scaling a novel therapy that could soon be filed for FDA approval.
The Man Behind the Mission: Dr. Sekar Kathiresan
Dr. Kathiresan’s trajectory from a working-class immigrant background to a leading figure in genomics is as compelling as his science.
A Harvard-trained cardiologist and former director of the Massachusetts General Hospital Center for Genomic Medicine, Kathiresan has published hundreds of papers on the genetic architecture of heart disease, identifying how common and rare variants contribute to lifelong risk.
But his most impactful decision came in 2018, when he left academia to found Verve Therapeutics, with a goal far more ambitious than typical biotech: make heart attacks optional.
“I lost my brother to a sudden heart attack in his early 40s,” he’s said. “I wanted to build something that could stop these tragedies before they happen—not just treat them after the fact.”
From Lab to Human Trials: A Fast Climb
VERVE-101 entered Phase 1 trials in 2022, enrolling patients with heterozygous familial hypercholesterolemia (HeFH), a genetic condition that causes dangerously high cholesterol. Early results were promising:
- Up to 65% reduction in LDL-C levels after a single dose
- Minimal adverse events
- No need for adjunct statin therapy in most cases
Now, Verve is expanding trials to broader populations, including those with established ASCVD but without genetic cholesterol disorders.
Lilly’s entry accelerates this timeline, with global Phase 2 trials slated for launch by Q1 2026 across the U.S., Europe, and Asia.
A New Class of “Cardio-Cures”
What makes Verve’s approach revolutionary is its preventive intent. Unlike most cardiology drugs that treat disease after it’s diagnosed, gene editing could intervene decades before the first symptom, reducing the lifetime risk of stroke and heart attack with a one-time therapy in early adulthood.
Verve is also working on:
- VERVE-102: A next-gen PCSK9 editor using an improved delivery system
- ANGPTL3 base editor: Targeting triglycerides and HDL metabolism
- Dual-targeting editors: One-shot treatments that silence multiple genes in parallel
Together, these represent a pipeline of “gene cures” for cardiovascular risk—what some are calling the “statin killers.”
Challenges Ahead: Ethics, Safety, and Scalability
Despite the excitement, hurdles remain. Base editing is still relatively new, and long-term safety data is limited. Off-target effects, durability beyond a few years, and equitable access remain open questions.
Dr. Kathiresan and Verve have been transparent about these concerns. The company is committed to:
- Post-marketing surveillance programs
- Diversity in trial recruitment, especially among underrepresented populations
- Partnerships with public health agencies to ensure affordability and access
There’s also the regulatory landscape: No in vivo base-editing therapy has yet received FDA approval. But Verve’s strong safety data and clear patient benefit could set a powerful precedent.
Market Implications: Disruption on a Genomic Scale
If Verve’s therapies gain approval, the implications for the pharmaceutical industry are seismic:
- Statin market erosion: A $15B annual category could shrink dramatically
- Employer interest: Payers may prefer one-time interventions to chronic prescriptions
- Health equity debate: Will gene editing become a tool for the rich—or a right for all?
Eli Lilly’s investment signals a paradigm shift—from disease management to molecular prevention.
What’s Next?
With the deal expected to close by late Q3 2025, Verve plans to:
- Finalize Phase 2/3 trial designs with global regulators
- Open a GMP manufacturing facility for lipid nanoparticle delivery
- Begin collaboration with Lilly’s cardiovascular team on next-gen therapeutics
- Explore pediatric trials for early-onset familial hypercholesterolemia
Meanwhile, investors, policymakers, and ethicists are watching closely, as Verve charts a future where editing your DNA may be safer than letting it run its natural course.
Conclusion: The Future of Heart Disease, Rewritten
Dr. Sekar Kathiresan has spent his career decoding the genetic code of heart disease. With Verve Therapeutics and the support of Eli Lilly, he’s now poised to rewrite it entirely.
If successful, Verve’s therapies could usher in a new age of one-time, curative medicine—where risk is removed before disease ever begins, and the world’s deadliest condition becomes, at last, preventable by design.