Will heart disease be cured with new gene therapy?
Here’s how one-shot PCSK9 gene base editing gene is predicted to lead to marked and durable reduction of LDL cholesterol
- May 26, 2026,
- Updated May 26, 2026 1:07 PM IST
A one-time gene-editing treatment being developed by Eli Lilly and Company is showing early signs of lowering harmful cholesterol levels, raising hopes of a new approach to managing cardiovascular risk. Heart disease remains among the leading causes of death globally, with coronary artery disease affecting more than 300 million people worldwide, according to the company.
The therapy, VERVE-102, is designed to switch off the PCSK9 gene linked to high LDL, or “bad”, cholesterol. In an early-stage study, a single infusion reduced LDL cholesterol by up to 62%, with the effect lasting for up to 18 months in some patients. The therapy also reduced circulating PCSK9 protein levels by up to 88%.
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The Phase 1b Heart-2 trial involved patients with inherited high cholesterol and premature coronary artery disease, conditions linked to elevated lifetime cardiovascular risk. The company said heterozygous familial hypercholesterolaemia, or HeFH, affects around one in every 200 to 250 people globally.
“These early data give us encouraging evidence that in vivo base editing of PCSK9 may offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment,” said Riyaz S. Patel, cardiologist at Barts Health NHS Trust and professor of cardiology at University College London.
The treatment is designed to mimic naturally occurring genetic variants associated with lifelong low cholesterol levels and lower risk of heart disease.
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“Twenty years ago, genetics showed us that people born with PCSK9 naturally turned off have low LDL-C for life and are remarkably protected from heart attack,” said Sekar Kathiresan, Lilly senior vice president and co-founder of Verve Therapeutics.
While statins and other cholesterol-lowering drugs are already widely used, they require lifelong treatment, making one-time gene-editing therapies a closely watched area for drugmakers and investors.
The company said no treatment-related serious adverse events were reported in the study involving 35 participants. Lilly plans to start Phase 2 studies later this year.
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A one-time gene-editing treatment being developed by Eli Lilly and Company is showing early signs of lowering harmful cholesterol levels, raising hopes of a new approach to managing cardiovascular risk. Heart disease remains among the leading causes of death globally, with coronary artery disease affecting more than 300 million people worldwide, according to the company.
The therapy, VERVE-102, is designed to switch off the PCSK9 gene linked to high LDL, or “bad”, cholesterol. In an early-stage study, a single infusion reduced LDL cholesterol by up to 62%, with the effect lasting for up to 18 months in some patients. The therapy also reduced circulating PCSK9 protein levels by up to 88%.
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The Phase 1b Heart-2 trial involved patients with inherited high cholesterol and premature coronary artery disease, conditions linked to elevated lifetime cardiovascular risk. The company said heterozygous familial hypercholesterolaemia, or HeFH, affects around one in every 200 to 250 people globally.
“These early data give us encouraging evidence that in vivo base editing of PCSK9 may offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment,” said Riyaz S. Patel, cardiologist at Barts Health NHS Trust and professor of cardiology at University College London.
The treatment is designed to mimic naturally occurring genetic variants associated with lifelong low cholesterol levels and lower risk of heart disease.
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“Twenty years ago, genetics showed us that people born with PCSK9 naturally turned off have low LDL-C for life and are remarkably protected from heart attack,” said Sekar Kathiresan, Lilly senior vice president and co-founder of Verve Therapeutics.
While statins and other cholesterol-lowering drugs are already widely used, they require lifelong treatment, making one-time gene-editing therapies a closely watched area for drugmakers and investors.
The company said no treatment-related serious adverse events were reported in the study involving 35 participants. Lilly plans to start Phase 2 studies later this year.
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