ScienceCTX310 gene-editing therapy reportedly shows cholesterol reductions at one year
A cardiologist reports about a 53% LDL cholesterol reduction at the highest dose in a 15-patient Phase 1a trial of CTX310. The account stresses that larger, longer studies are needed and the therapy is not FDA-approved.
TLDR
A cardiologist describes CTX310 as a CRISPR-Cas9 therapy delivered through one IV infusion to switch off ANGPTL3 in the liver. The account describes a 15-patient Phase 1a trial involving people with difficult-to-treat lipid disorders who were already receiving standard care. At the highest dose, reported reductions at one year were about 79% for ANGPTL3, 53% for LDL cholesterol and 48% for triglycerides. The cardiologist reports no treatment-related serious adverse events or dose-limiting toxicities during that follow-up, but emphasizes the limits: a tiny early trial, no hard cardiovascular outcomes yet and no FDA approval. Safety and durability still need to be established in larger, longer studies.