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Verve Therapeutics to Kick Off US Trial for Base-Editing Therapy After FDA Lifts Hold, Clears IND

NEW YORK – The US Food and Drug Administration has given Verve Therapeutics permission to begin enrolling US patients into the Phase Ib trial for its gene-editing therapy VERVE-101, an investigational treatment for heterozygous familial hypercholesterolemia (HeFH), the biotechnology company said Monday.

The FDA last November placed Verve's investigational new drug (IND) application for VERVE-101 on hold, due to questions about potency differences between human and nonhuman cells and concerns about germline editing and off-target effects. With the hold lifted and the company's IND cleared, Verve can now conduct a clinical trial in the US.

"This clearance, for the first time, enables clinical development of an in vivo base-editing product candidate in the United States," Verve CSO Andrew Bellinger said in a statement.

HeFH is an inherited disease in which lifelong elevations in blood low-density lipoprotein cholesterol can lead to heart attack, stroke, and accelerated atherosclerotic cardiovascular disease. VERVE-101, an in vivo base-editing therapy, is designed to permanently inactivate the PCSK9 gene in the liver in an effort to lower LDL-C.

Boston-based Verve submitted interim clinical data from the dose-escalation portion of its ongoing Phase Ib trial in the UK and New Zealand to address the FDA's questions. The open-label Phase Ib trial, dubbed heart-1, is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of VERVE-101 in patients with HeFH.