By Fineline Cube 
Shanghai-based YolTech Therapeutics announced that it has received clearance from the National Medical Products Administration (NMPA) to carry out a clinical study for its YOLT-101 in heterozygous familial hypercholesterolemia (HeFH). This follows the FDA’s approval for trials in June 2025, making YOLT-101 China’s first in vivo base-editing therapy to receive clinical approvals in both China and the United States.
Innovative Base-Editing Technology
YOLT-101 is a next-generation in vivo base-editing drug independently developed by YolTech. It features a proprietary adenine base editor (ABE) YolBE (specifically hpABE5), engineered by fusing nCas with a novel deaminase derived from Hafnia paralvei, and novel lipid nanoparticles (LNP) as the delivery vehicle. Unlike conventional CRISPR/Cas9, which induces DNA double-strand breaks, YolBE enables single-base precision editing without DNA cleavage, significantly reducing risks of chromosomal abnormalities and off-target effects.
Mechanism of Action
By precisely editing the PCSK9 gene at the DNA level, YOLT-101 silences PCSK9 expression, lowers circulating PCSK9 protein levels, inhibits PCSK9-mediated degradation of LDL receptors (LDLR), enhances LDLR-mediated uptake of low-density lipoprotein (LDL), and effectively clears LDL cholesterol (LDL-C) from blood. This mechanism has the potential to halt or reverse the progression of HeFH.-Fineline Info & Tech