Gene Therapy for Cholesterol: Initial Test Results Cheer

Eli Lilly working on single-dose drug to be administered via IV for long-term gain

Mumbai: For millions of heart patients struggling with elevated levels of cholesterol, there is a ray of hope, A one-time intravenous infusion of a drug coded VERVE-102, being tested by global pharmaceutical giant Eli Lilly, showed significant reductions in a liver-produced protein called PCSK9.

VERVE-102 is an early investigational in vivo (inside the body) gene editing medicine designed to make a single, precise
DNA base pair change to PCSK9 gene. It is created to mimic naturally occurring cardioproteсtive PCSK9 variants and lower
LDL-C (‘bad’ cholesterol) over time, with a single infusion.

The US Food and Drug Administration has granted fast track designation for VERVE-102 to reduce LDL-C in participants with hyper lipidaemia and high lifetime cardiovascular risk. In India, 60-80 million are estimated to be living with heart conditions, while reports suggest three million cases of heart attacks annually.

“In an interim analysis of 35 patients, a single infusion of VERVE-102 resulted in dose-dependent mean reductions in
PCSK9, ranging from 51% to 88%, at 0.3 mg/kg or 1.0 mg/kg. respectively Corresponding reductions in LDL-C ranged from
9% to 62%, at 0.3 mg/kg or 1.0 mg/kg, respectively, “an Eli Lilly India spokesperson told ET.

“These early data give us encouraging evidence,” said Riyaz S Patel, cardiologist at Barts Hеalth NHS Trust and professor of cardiology at University College London. “Many patients with elevated LDL-C struggle to achieve sustained control… putting them at significant risk. With coronary artery disease still one of the leading causes of death worldwide, the need for new approaches is real.”

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