In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial
Study context
- Population
- Adults with heterozygous familial hypercholesterolemia and uncontrolled low-density lipoprotein cholesterol (LDL-C)3 men · 3 women
- Participants
- 6 adults
- Comparison
- Escalating single doses of YOLT-101
- 0.6 mg kg-1n = 3
- Outcome
- Safety and tolerability
Methodological detailsdose-escalation, single intravenous dose, ongoing, interim analysis
- Dose-escalation
- Single intravenous dose
- Ongoing
- Interim analysis
Main finding
As reported in the abstract
- No grade ≥3 adverse events
- Most common AEs: transient, self-limited infusion-related reactions and liver enzyme elevations
- Interim analysis
Further findings
- Reduction in circulating PCSK9 and LDL-C (0.6 mg kg-1 cohort) · 24 weeks
- 74.4% · PCSK9 reduction · LDL-C reduction 52.3%
Reduction in circulating PCSK9 and LDL-C (0.6 mg kg-1 cohort) · 24 weeks
- Dose-dependent and durable reductions in PCSK9 and LDL-C after a single infusion
Safety
- No grade ≥3 adverse events
- Transient, self-limited infusion-related reactions and liver enzyme elevations were the most common AEs
- Interim analysis
Original publication
In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial. Nat Med. Published 3 March 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →