YolTech Therapeutics Announces Favorable Interim Results from Ongoing IIT that Single-Dose Administration of YOLT-101, an In Vivo Gene Editing Therapy, Effectively Reduces LDL-C in Familial Hypercholesterolemia

Shanghai, China - March 26, 2025 -- YolTech Therapeutics Co.,Ltd, a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines, announced promising interim clinical data from an investigator-initiated trial (IIT) of YOLT-101 (NCT06458010). YOLT-101 is an investigational in vivo base editing therapy for the treatment of heterozygous familial hypercholesterolemia (HeFH). Patients with HeFH have elevated low-density lipoprotein cholesterol (LDL-C) levels and premature atherosclerotic cardiovascular disease. YOLT-101 is designed to reduce the LDL-C levels in HeFH patients through editing of PCSK9 gene, which enhances low-density lipoprotein receptor (LDLR)-mediated uptake of LDL particles.

YOLT-101-IIT is an open-labeled, single-center, dose escalation study with Renji Hospital affiliated to Shanghai Jiao Tong University to evaluate the safety, tolerability, preliminary efficacy and pharmacodynamics profile of YOLT-101 in patients with heterozygous familial hypercholesterolemia (HeFH).

Single dose of YOLT-101 led to dose-dependent reduction of low-density lipoprotein cholesterol (LDL-C) during 16-week primary observation period. As early as one-week post-dosing, reduction of LDL-C levels was observed in the mid- and high-dose cohorts. At four-week post-dosing, LDL-C level was reduced by up to 70% in the high-dose cohort, underscoring YOLT-101’s potential to lower LDL-C levels in HeFH patients.

YOLT-101 was well-tolerated in all dose levels. As of January 31, 2025, no serious adverse events (SAE), dose-limiting toxicities (DLTs), or CTCAE v5.0 grade ≥3 adverse events were observed across all dose cohorts. There was no patient discontinuations due to adverse events.

"We are committed to revolutionizing traditional approaches to cardiovascular disease through next-generation base editing technology and groundbreaking single-dose treatment," said Dr. Yuxuan Wu, Founder and CEO of YolTech Therapeutics. "Data from YOLT-101-IIT strongly suggests a single YOLT-101 administration can lead to significant and durable reductions in LDL-C levels for at least six months. It benefits long-term LDL-C level control in FH patients as conventional treatments require long-term patient adherence during years or even decades of medications. By precisely editing key genes regulating LDL-C levels in the liver, YOLT-101 has the potential to be a transformative ‘one-time treatment, lifelong cure’ therapy. We look forward to further supporting its safety and efficacy in larger clinical studies and expediting its global development to benefit patients worldwide.”

About Familial Hypercholesterolemia (FH)

Familial hypercholesterolemia (FH) is caused by mutations in LDL metabolism-related genes (LDLR, APOB, PCSK9). As a results, patients with FH have markedly elevated low-density lipoprotein cholesterol (LDL-C) levels and premature atherosclerotic cardiovascular disease (ASCVD). The global prevalence of FH is 1:200-1:250, affecting a total of ~34 million people worldwide. 

Current FH management strategies include lifestyle modifications, lipid-lowering medications, lipoprotein apheresis, and liver transplantation. The standard of treatment involves pharmacotherapy, with most patients receiving a triple regimen of statins, ezetimibe, and PCSK9 inhibitors. However, current therapies have significant limitations including suboptimal efficacy, intolerable side effects, and poor long-term adherence. These challenges underscore the urgent need for novel, durable therapies to address the lifelong treatment burden of FH.

About YolTech

YolTech Therapeutics is a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines. Our approach combines innovative gene editing technologies with an advanced lipid nanoparticle (LNP) delivery system, creating a versatile platform designed to address a wide range of serious diseases. Central to our mission is the development of internal capabilities, including end-to-end manufacturing, to ensure the highest standards of quality and scalability. Our lead candidate, targeting ATTR, marks a significant milestone as China's first LNP-mediated in vivo gene editing therapy to enter clinical development. With promising early clinical outcomes, YolTech is also advancing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1). As a company dedicated to transforming the treatment landscape, YolTech continues to push the boundaries of what is possible in gene editing. 

For more information, please visit: www.yoltx.com