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
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; only low-grade infusion reactions and fatigue were reported.
“These early data give us encouraging evidence that in vivo base editing of PCSK9 may offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment,” said Riyaz S. Patel, cardiologist at Barts Health NHS Trust and professor of cardiology at University College London (UK). “Many patients with elevated LDL-C struggle to achieve sustained control despite ongoing efforts with the medicines available today, putting them at significant risk for cardiovascular events. With coronary artery disease still one of the leading causes of death worldwide, the need for new approaches is real.”
UCLA expands cell and gene therapy production capabilities
UCLA opened its Center for Advanced Biotherapies, a 14,000-square-foot FDA-compliant manufacturing facility that nearly doubles the institution’s capacity to produce personalized cell and gene therapies for clinical trial patients. Built with US$7.3 million from the National Institutes of Health and US$2 million from the California Institute for Regenerative Medicine, the facility can produce up to 150 therapy products annually.
The center features 10 cleanrooms, including seven manufacturing suites for simultaneous therapy production, two bioengineering rooms with bioreactors and 3D printers, and a dedicated viral vector manufacturing suite. Its location within UCLA’s Center for Health Sciences enables same-day delivery from manufacturing to patient bedside.
“The [Center for Advanced Biotherapies] doesn’t just do one thing,” said Robert Prins, a tumor immunologist and professor of neurosurgery and pharmacology. “It does dendritic cell vaccines for brain tumor patients, engineered T cells for melanoma and sarcoma patients, bispecific CAR-T cells for lymphoma patients and gene therapy for SCID patients. You’re treating all different types of diseases, in kids and adults, at this one center — that’s what distinguishes it.”
VectorBuilder announces plans for Chicago-based R&D and biomanufacturing center
VectorBuilder announced it is investing US$50 million to establish an advanced biomanufacturing and research and discovery center in Chicago. The multi-phase facility is expected to begin operations in 2027, with expansion based on customer demand.
The center will integrate research, development and manufacturing capabilities across VectorBuilder’s gene delivery portfolio, including plasmids, recombinant viral and nonviral vectors and genetically engineered cells. This unified approach aims to streamline the transition from research-grade constructs to GMP manufacturing, reducing development fragmentation.
The Chicago center will complement VectorBuilder’s existing global operations across the United States, Europe and Asia, strengthening regional support for researchers and biopharmaceutical companies advancing toward clinical and commercial stages.
“Despite recent volatility in the CGT field, we see strong long-term momentum in genetic medicine,” said Bruce Lahn, Chief Scientist of VectorBuilder. “This investment reflects our conviction that gene delivery will be a defining factor in the next phase of the industry. By expanding our capabilities, we can tap into local talent pools and strengthen our biomanufacturing and R&D capabilities in a fully integrated manner, positioning us to innovate and scale as demand accelerates.”