In three people with inherited high cholesterol, one injection of the gene therapy lowered PCSK9 and LDL cholesterol for at least six months, indicating the edit may last a long time.
Evidence from Studies
No evidence studies found yet.
What Would Prove This
Per GRADE and EBM methodology, here is what ideal scientific evidence would look like to definitively prove or disprove this claim, ordered from strongest to weakest.
Whether YOLT-101 or similar base-editing therapies produce sustained LDL-C reductions beyond 1 year across multiple trials and whether effects plateau or decline over time.
A systematic review and meta-analysis of all published trials of YOLT-101 or similar therapies, pooling LDL-C and PCSK9 levels at 12, 24, 52, and 104 weeks to determine the trajectory of reduction and stability of effect.
Whether YOLT-101 produces sustained LDL-C reduction compared to placebo over 1 year.
A double-blind, placebo-controlled trial of 100 adults with heterozygous familial hypercholesterolemia, randomized to YOLT-101 (0.6 mg/kg) or placebo, with LDL-C and PCSK9 measured monthly for 12 months and liver biopsies at 12 months to assess editing persistence.
Whether LDL-C levels remain reduced for 5–10 years after a single YOLT-101 infusion in real-world settings.
A prospective cohort study of 200 individuals treated with YOLT-101, with annual LDL-C, PCSK9, and liver function testing for 10 years, and periodic NGS analysis of liver tissue to quantify editing persistence.
Whether individuals with persistent LDL-C reduction after YOLT-101 have higher rates of on-target editing in liver tissue compared to those with partial response.
A case-control study comparing liver biopsy samples from 20 individuals with >50% LDL-C reduction at 24 months to 20 with <20% reduction, quantifying A-to-G editing efficiency at the PCSK9 target site via deep sequencing.
Whether individuals treated with YOLT-101 more than 6 months ago have lower average LDL-C than untreated individuals with the same condition.
A cross-sectional analysis of 300 adults with heterozygous familial hypercholesterolemia, comparing LDL-C levels between those treated with YOLT-101 (≥6 months prior) and those on standard therapy, adjusting for age, sex, and baseline LDL-C.