Overview
- The late-breaking Phase 1 results, presented at the AHA Scientific Sessions 2025, tested a one-time CTX310 infusion in 15 adults in Australia, New Zealand, and the U.K. with at least 60 days of follow-up.
- At the highest prespecified dose, mean LDL fell about 50% and triglycerides about 55%, with levels remaining reduced through at least 60 days after a roughly two-week onset.
- Reporting from the full cohort indicates peak reductions approaching 80% in some participants at higher doses, a finding that will require confirmation in larger trials.
- Safety findings to date include mild infusion-related symptoms and one transient rise in liver enzymes, with no serious adverse events reported and long-term monitoring planned for up to 15 years.
- Investigators plan Phase 2 studies in late 2025 or early 2026, noting the small, primarily male cohort and unresolved questions about durability beyond 60 days, clinical outcomes, and real-world access.