This Week’s CGT News: Base Editing Takes Aim at Cholesterol
Verve reports phase 1 data for one-time PCSK9 base editing; plus: neuroprotective gene therapy, non-viral DNA delivery, tumour-activated CAR-T, and in vivo CAR-M
Clinical Scorecard: This Week’s CGT News: Base Editing Takes Aim at Cholesterol
At a Glance
Category Detail
Condition Heterozygous familial hypercholesterolemia or premature coronary artery disease Key Mechanisms Inactivation of PCSK9 in the liver using adenine base editing Target Population Adults with heterozygous familial hypercholesterolemia or premature coronary artery disease Care Setting Phase 1 clinical trial
Key Highlights
VERVE-102 showed up to 88% reduction in PCSK9 and 62% reduction in LDL-C No dose-limiting toxicities reported in the study Mild-to-moderate infusion reactions and transient liver enzyme elevations observed Effects appeared durable through follow-up of at least one year in 15 participants Guideline-Based Recommendations
Diagnosis
Management
Monitoring & Follow-up
Risks
Mild-to-moderate infusion reactions Transient liver enzyme elevations Patient & Prescribing Data
35 participants in an open-label, single-ascending-dose study
One-time treatment delivered via lipid nanoparticle
Clinical Best Practices
Monitor liver enzyme levels post-treatment Assess for infusion reactions during administration Related Resources & Content
