Pharma Focus Asia

Initial Dosing of Patient Commences in Clinical Study Investigating YOLT-101 for FH Therapy

Wednesday, April 03, 2024

YolTech Therapeutics has announced a significant milestone in its development of YOLT-101, an in vivo genome editing candidate for treating Familial Hypercholesterolemia (FH). The company has initiated an Investigator-Initiated Trial (IIT) by dosing the first patient with YOLT-101.

FH is a genetic disorder affecting the body's ability to remove low-density lipoprotein (LDL) cholesterol from the bloodstream, leading to an increased risk of early heart disease.

YOLT-101, developed independently by YolTech, targets specific genetic mutations associated with FH using advanced gene editing technology and lipid nanoparticle (LNP) delivery. Preclinical studies in non-human primate (NHP) models have shown significant and sustained reduction in LDL-C levels for nearly two years after a single dose.

The clinical trial initiated by investigators is a single-arm, open-label, dose-escalation study. Its primary objectives include assessing the safety and tolerability of YOLT-101, determining the Optimal Biologically Active Dose (OBD), and preliminarily evaluating its impact on lipid and lipoprotein levels in FH patients.

Dr. Yuxuan Wu, Founder and CEO of YolTech, expressed optimism about the potential of YOLT-101 to revolutionize treatment for FH patients. YolTech is committed to advancing the clinical development of YOLT-101 and collaborating closely with regulatory authorities, healthcare professionals, and patient advocacy groups.

YOLT-101 is an in vivo liver-based editing therapy designed to permanently deactivate the PCSK9 gene, thereby reducing disease-driving LDL-C levels. It is currently undergoing evaluation in the IIT clinical trial for patients with high-risk heterozygous FH, established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled LDL-C levels on standard-of-care therapy.



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