By Chris Wack
Verve Therapeutics said Monday that the clinical hold and clearance of its Investigational New Drug application by the U.S. Food and Drug Administration has been lifted, allowing it to conduct a clinical trial in the U.S. evaluating VERVE-101 for the treatment of heterozygous familial hypercholesterolemia.
VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood low-density lipoprotein cholesterol.
The biotechnology company said it submitted interim clinical data from the dose-escalation portion of the ongoing heart-1 Phase 1b clinical trial and addressed the FDA’s preclinical questions in its response to the clinical hold.
The heart-1 trial is evaluating the safety, tolerability, pharmacokinetic and pharmacodynamic profile of VERVE-101 in patients with HeFH, and is currently being conducted at sites in the U.K. and New Zealand.
Write to Chris Wack at [email protected]
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