Gene editing potential treatment for hereditary killer

pharmafile | July 13, 2022 | News story | Research and Development  

Verve therapeutics has dosed the first patient win its heart-1 clinical trial, studying the safety and efficacy of its in vivo base editing medicine, VERVE-101. The novel investigative medicine is being researched as a potential treatment for heterozygous familial hypercholesterolemia.

Verve is a biotechnology company working in new approaches to the care of cardiovascular disease with single-course gene editing medicines.  VERVE-101 is designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver, to reduce disease-driving low-density lipoprotein cholesterol (LDL-C).

The trial will evaluate VERVE-101 as a treatment for patients with heterozygous familial hypercholesterolemia (HeFH), a prevalent and potentially life-threatening subtype of atherosclerotic cardiovascular disease (ASCVD).

“The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing,” said Sekar Kathiresan, MD, co-founder and chief executive officer of Verve. “Preclinical data suggest that VERVE-101 has the potential to offer people with HeFH a game-changing treatment option, transforming the traditional chronic care model to a single-course, life-long treatment solution.”

The heart-1 clinical trial will enrol approximately 40 adult HeFH patients with established ASCVD and evaluate the safety and tolerability of VERVE-101 administration, with additional analyses for pharmacokinetics and reductions in blood PCSK9 protein and LDL-C.

“Our ultimate goal with VERVE-101 is to bring a new option to the millions of people with ASCVD around the world, and dosing participants in the Phase 1 study for this first indication, HeFH, is a key inflection point to achieving that goal,” said Andrew Bellinger, M.D., Ph.D., chief scientific and medical officer of Verve. “With the current standard of care treatment for HeFH, less than 20% of patients achieve LDL-C goal levels due to the limitations of the chronic model which requires rigorous patient adherence, regular health care access, and extensive health care infrastructure. VERVE-101 has the potential to change the way cardiovascular disease is cared for by lowering LDL-C as low as possible for as long as possible after a single treatment.”

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