YolTech Therapeutics has received the US Food and Drug Administration (FDA) approval for its investigational new drug (IND) application for YOLT-101 to treat heterozygous familial hypercholesterolemia (HeFH).
YOLT-101 is an in vivo base editing therapy designed to target proprotein convertase subtilisin/kexin type 9 (PCSK9).
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Its innovative approach lies in its potential to durably reduce blood low-density lipoprotein cholesterol (LDL-C) levels through a single-dose treatment.
The technology behind YOLT-101 incorporates YolTech’s adenine base editor, known as hpABE5, which consists of nCas and a new deaminase evolved from Hafnia paralvei.
To deliver this therapeutic agent, the company employs an advanced lipid nanoparticle (LNP) delivery system.
hpABE5 can perform precise A•T to G•C base conversions without inducing DNA double-strand breaks, thus minimising risks associated with chromosomal abnormalities and off-target effects.
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By GlobalDataYolTech Therapeutics co-founder and CEO Yuxuan Wu stated: “The FDA IND clearance marks a significant milestone for YolTech. In vivo gene editing represents a new generation of therapeutics — offering one-time, durable solutions for chronic and genetic diseases.
“We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients living with severe genetic and cardiovascular diseases.”
Currently under evaluation in an ongoing investigator-initiated trial (IIT), YOLT-101 has shown promising results with a favourable safety profile and substantial LDL-C–lowering effects.
Familial hypercholesterolemia is a genetic disorder stemming from mutations affecting LDL metabolism-related genes such as LDLR, Apolipoprotein B (APOB) and PCSK9.
Individuals suffering from FH typically experience elevated LDL-C levels, which contribute to a heightened risk of developing early-onset atherosclerotic cardiovascular diseases.
