Enrolment in a clinical trial of a gene editing therapy for patients with heterozygous familial hypercholesterolemia has been paused after laboratory abnormalities were detected in one participant. The therapy, VERVE-101, aims to permanently turn off the PCSK9 gene to reduce LDL cholesterol levels. Initial results showed promising reductions in LDL cholesterol, but one participant experienced a grade 3 increase in liver enzymes after treatment. Despite this, the company behind the therapy, Verve Therapeutics, plans to investigate the issue further and work with regulatory authorities to move forward. They are also prioritizing a new trial, VERVE-102, with a different delivery system.
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Heart-1 Gene Therapy Trial Pauses Enrollment
