Verve Therapeutics Announces Interim Data for VERVE-101 Demonstrating First Human Proof-of-Concept for In Vivo Base Editing with Dose-Dependent Reductions in LDL-C and Blood PCSK9 Protein in Patients with Heterozygous Familial Hypercholesterolemia
BOSTON, Nov. 12, 2023 (GLOBE NEWSWIRE) -- Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced first human proof-of-concept data for in vivo base editing from the ongoing heart-1 phase 1b clinical trial of VERVE-101. Treatment with VERVE-101 led to dose-dependent reductions of disease-causing low-density lipoprotein cholesterol (LDL-C) in people living with heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterized by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). 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 LDL-C.
- The data showed that VERVE-101 could meaningfully and durably lower LDL-C in these patients.
- The trial is designed to evaluate the safety and tolerability of VERVE-101, with additional analyses for pharmacokinetics and pharmacodynamic reductions in blood PCSK9 protein and LDL-C.
- Initial safety data reported are from all ten patients enrolled as of a data cut-off date of October 16, 2023.
- VERVE-102 is an in vivo base editing medicine that aims to inactivate the PCSK9 gene in a similar way to VERVE-101.