SHANGHAI, China I April 2, 2024 I YolTech Therapeutics announced that the first patient has been dosed with YOLT-101, the company’s in vivo genome editing candidate being developed as a single dose, potentially curative therapy for Familial Hypercholesterolemia(FH), marking the commencement of an Investigator-Initiated Trial (IIT).

Familial Hypercholesterolemia (FH) is a genetic disorder that affects the body’s ability to remove low-density lipoprotein (LDL) cholesterol from the blood. It is characterized by high levels of LDL cholesterol, also known as “bad” cholesterol, which can lead to an increased risk of early heart disease.

YOLT-101, developed independently by YolTech, aims to intervene in specific genetic mutations associated with Familial Hypercholesterolemia. Leveraging advanced gene editing technology and lipid nanoparticle (LNP) delivery, YOLT-101 promises to provide effective treatment options for patients. The preclinical data of YOLT-101 indicate that a single dose of the drug resulted in a significant reduction of LDL-C levels lasting nearly two years in non-human primate (NHP) models.

The clinical study of YOLT-101, initiated by investigators, is a single-arm, open-label, dose-escalation clinical trial. This trial’s primary objectives are to evaluate its safety, tolerability,  as well as the identification of an Optimal Biologically Active Dose (OBD) in patients with Familial Hypercholesterolemia, and preliminarily assess the impact of a single dose of YOLT-101 on lipid and lipoprotein levels in plasma.

“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.” said Dr. Yuxuan Wu, Founder and CEO of YolTech, “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.”

YolTech remains dedicated to advancing the clinical development of YOLT-101 and collaborating closely with regulatory authorities, healthcare professionals, and patient advocacy groups to contribute to the progress of gene editing therapies.

About YOLT-101

A single-course in vivo liver base editing medicine, is currently being evaluated in our IIT clinical trial in patients with high-risk heterozygous familial hypercholesterolemia (FH), established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled LDL-C levels on oral standard-of-care therapy. These single-course treatment is designed to permanently turn off the PCSK9 gene in the liver to reduce disease-driving LDL-C.

About YolTech

YolTech Therapeutics is a pioneering gene editing company dedicated to develop a robust gene editing medicines to treat patients with serious diseases, which has built leading high-throughput evolution platform and innovative LNP deliver system. It possesses strong capability of novel Cas and base editor discovery and exceptional in-house LNP production capacity for GMP manufactory, with independent intellectual property rights and core patent protection globally. It has created a pipeline with 10+ genetic medicines focusing on cardiovascular diseases, metabolic diseases, infectious diseases as well as more common and rare diseases. Clinical trial clearance has been received for its leading in vivo gene editing asset YOLT-201 in March 2024.

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SOURCE: YolTech Therapeutics