Cell Therapy Weekly: Base Editing Therapy for Hypercholesterolemia

This week: Eli Lilly (IN, USA) shared positive results from its Phase Ib trial investigating its single-dose base editing therapy for hypercholesterolemia, the University of California, Los Angeles (UCLA; CA, USA) opened its Center for Advanced Biotherapies, and VectorBuilder (IL, USA) announced it is investing US$50 million to establish an advanced biomanufacturing and research and discovery center in Chicago. The news highlights: Single-dose gene therapy shows promise for lowering cholesterol UCLA expands cell and gene therapy production capabilities VectorBuilder announces plans for Chicago-based R&D and biomanufacturing center

Cell Therapy Weekly: Base Editing Therapy for Hypercholesterolemia. Single-dose gene therapy shows promise for lowering cholesterol Eli Lilly and Company announced positive Phase Ib Heart-2 study results for VERVE-102, an investigational in vivo base editing therapy designed to permanently turn off the PCSK9 gene and lower low-density lipoprotein cholesterol (LDL-C) with a single infusion. The trial is evaluating VERVE-102 in adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. A single intravenous dose produced dose-dependent PCSK9 reductions of 51-88% and LDL-C reductions ranging from 9% to 62% across six dose levels (0.3-1.0 mg/kg). These reductions remained sustained for up to 18 months. VERVE-102 was well tolerated with no serious treatment-related adverse events or dose-limiting toxicities; on...