Gene-Editing Therapy Shows Promise for Cholesterol Reduction in Early Trial
- Verve Therapeutics reported Phase 1 trial results showing their base editing gene therapy reduced LDL cholesterol by up to 55% in patients with familial hypercholesterolemia.
- The treatment uses base editing to modify the PCSK9 gene in liver cells, potentially offering a one-time therapy to replace daily cholesterol medications.
- While promising, the 10-patient trial raises safety concerns due to permanent DNA changes, requiring extensive long-term follow-up before broader approval.
- Experts emphasize the need for larger studies and years of additional research before this personalized approach could become widely available.