The therapy is delivered through an IV drip using nanoparticles that specifically carry gene-editing tools to liver cells, avoiding the need to remove and reinfuse cells.
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 GalNAc-modified lipid nanoparticles consistently achieve hepatocyte-specific delivery and gene editing in vivo across different gene targets and editing platforms.
A systematic review and meta-analysis of all published human trials using GalNAc-LNP for in vivo gene editing, comparing hepatocyte delivery efficiency, off-target editing, and immune responses across different payloads (base editors, CRISPR-Cas9, siRNA).
Whether GalNAc-LNP delivery of YOLT-101 achieves higher hepatocyte editing efficiency than alternative delivery systems.
A double-blind, randomized trial of 60 adults with heterozygous familial hypercholesterolemia, comparing YOLT-101 delivered via GalNAc-LNP versus the same therapy delivered via non-targeted LNP, with liver biopsy at 4 weeks to quantify editing efficiency via NGS.
Whether individuals with pre-existing liver disease have reduced hepatocyte delivery or editing efficiency after YOLT-101 administration.
A prospective cohort study of 150 individuals with heterozygous familial hypercholesterolemia, stratified by liver fibrosis stage (F0–F4), measuring editing efficiency via liver biopsy and PCSK9 reduction over 12 months.
Whether individuals with poor response to YOLT-101 have lower expression of the asialoglycoprotein receptor (ASGPR) on hepatocytes compared to responders.
A case-control study comparing ASGPR expression in liver biopsy tissue from 20 responders (≥50% LDL-C reduction) and 20 non-responders (<20% reduction) using immunohistochemistry and RNA sequencing.
Whether GalNAc-LNP delivery of YOLT-101 results in detectable editing in liver tissue at a single time point.
A cross-sectional analysis of 30 individuals treated with YOLT-101, measuring editing efficiency in liver biopsy tissue at 4–8 weeks post-infusion via deep sequencing.