In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial - PubMed
3 hours ago
- #gene therapy
- #familial hypercholesterolemia
- #base editing
- Phase 1 trial evaluates YOLT-101, an in vivo base-editing gene therapy for heterozygous familial hypercholesterolemia.
- YOLT-101 uses adenine base-editing technology delivered via GalNAc-modified lipid nanoparticles to target PCSK9.
- Six participants received escalating doses (0.2, 0.4, or 0.6 mg/kg) with no grade ≥3 adverse events reported.
- Most common adverse events were transient infusion-related reactions and liver enzyme elevations.
- Dose-dependent reductions in PCSK9 (74.4%) and LDL-C (52.3%) were sustained at 24 weeks in the highest dose cohort.
- Study demonstrates potential for durable LDL-C reduction with a single infusion of YOLT-101.