YolTech doses first subject in familial hypercholesterolemia trial

The trial will evaluate the preliminary impact of the drug on plasma lipid and lipoprotein levels.

Vishnu Priyan April 03 2024

YolTech Therapeutics has dosed the first subject in an investigator-initiated trial (IIT) of YOLT-101, an in vivo genome editing candidate for familial hypercholesterolemia (FH).

The open-label, single-arm, dose-escalation trial aims to establish the safety and tolerability of YOLT-101, and also identify an optimal biologically active dose (OBD).

It will also evaluate the preliminary impact of the drug on plasma lipid and lipoprotein levels.

YOLT-101 uses advanced gene editing technology and lipid nanoparticle (LNP) delivery systems to target specific genetic mutations responsible for FH.

Preclinical data have shown that a single dose of YOLT-101 can significantly reduce LDL-C levels for up to two years in non-human primate models.

A genetic disorder, FH impairs the body's ability to remove low-density lipoprotein (LDL) cholesterol from the blood.

YOLT-101 is designed as a one-time, in vivo liver base editing treatment. It aims to permanently deactivate the PCSK9 gene in the liver, which is expected to lower the LDL-C levels that drive the disease.

The trial includes patients with high-risk heterozygous FH, established atherosclerotic cardiovascular disease, and uncontrolled LDL-C levels despite oral standard-of-care therapy.

YolTech founder and CEO Dr Yuxuan Wu said: “The successful administration of YOLT-101 marks a significant breakthrough for in vivo gene editing therapies in this field. We anticipate YOLT-101 delivering unprecedented clinical outcomes for patients with familial hypercholesterolemia.

“We extend our gratitude to all researchers, partners, and supporters involved in the YOLT-101 project and we anticipate that YOLT-101 will offer new treatment options for patients.”

The latest development comes after the company dosed the first subject with YOLT-201 in the Phase I trial for treating hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

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