In six people with inherited high cholesterol, one injection of the experimental therapy caused temporary increases in liver enzymes and mild flu-like symptoms after infusion, but no serious side effects occurred.
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 consistently cause transient liver enzyme elevations or infusion reactions across multiple trials and whether these are predictable or dose-dependent.
A systematic review and meta-analysis of all published clinical trials of YOLT-101 or similar GalNAc-LNP delivered base editors, pooling incidence and severity of infusion reactions and ALT/AST elevations across all dose levels and follow-up durations.
Whether YOLT-101 causes transient liver enzyme elevations compared to placebo in a controlled setting.
A double-blind, placebo-controlled trial of 150 adults with heterozygous familial hypercholesterolemia, randomized to YOLT-101 (0.6 mg/kg) or saline placebo, with daily liver enzyme monitoring for 14 days and weekly monitoring for 12 weeks, and standardized assessment of infusion reactions.
Whether transient liver enzyme elevations after YOLT-101 are associated with long-term liver function decline or fibrosis.
A prospective cohort study of 300 individuals treated with YOLT-101, with annual liver elastography, ALT/AST, and FibroScan measurements for 10 years, compared to 300 matched controls on standard therapy.
Whether individuals who develop persistent liver enzyme elevations after YOLT-101 have distinct genetic or immunological profiles compared to those who do not.
A case-control study comparing 50 individuals with persistent ALT elevation (>2x ULN at 12 weeks) after YOLT-101 to 100 individuals with normal ALT, analyzing pre-treatment immune markers, HLA types, and lipid nanoparticle metabolism genes.
Whether liver enzyme levels at a single time point are higher in individuals treated with YOLT-101 compared to untreated individuals with the same condition.
A cross-sectional analysis of 500 adults with heterozygous familial hypercholesterolemia, comparing median ALT and AST levels between those who received YOLT-101 (at least 4 weeks prior) and those on statins or PCSK9 inhibitors alone.