YolTech Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for YOLT-101, an In Vivo Base Editing Therapy to Treat Heterozygous Familial Hypercholesterolemia (HeFH)

SHANGHAI, June 5, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage biotechnology company developing in vivo genome editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application for YOLT-101, an in vivo base editing therapy targeting PCSK9 for the treatment of heterozygous familial hypercholesterolemia (HeFH).

"The FDA IND clearance marks a significant milestone for YolTech," said Dr. Yuxuan Wu, Co-founder and CEO of YolTech Therapeutics. "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. ''

About YOLT-101

YOLT-101 is an investigational in vivo base editing therapy designed to be a single-dose treatment to durably reduce blood LDL-C. It is based on YolTech's proprietary adenine base editor, YolBE—specifically hpABE5—which comprises nCas and a novel deaminase evolved from Hafnia paralvei. For delivery, YOLT-101 utilizes YolTech's innovative lipid nanoparticle (LNP) delivery system. Unlike traditional CRISPR/Cas9 systems that rely on DNA double-strand breaks (DSBs), hpABE5 enables precise A•T to G•C base conversion without introducing DSBs, thereby significantly reducing risks of chromosomal abnormalities and off-target effects. YOLT-101 is being evaluated in ongoing investigator-initiated trial (IIT), where it has demonstrated a favorable safety profile and robust LDL-C–lowering effects. For detailed clinical data, please refer to: https://doi.org/10.1101/2025.04.17.25325983

About Familial Hypercholesterolemia (FH)

Familial hypercholesterolemia (FH) is caused by mutations in LDL metabolism-related genes such as LDLR, APOB, and PCSK9. Patients with FH exhibit significantly elevated levels of low-density lipoprotein cholesterol (LDL-C) and a high risk of early-onset atherosclerotic cardiovascular disease (ASCVD). The estimated global prevalence of FH is 1 in 200 to 1 in 250, affecting approximately 34 million individuals worldwide. Current treatment strategies—lifestyle modifications, statins, ezetimibe, PCSK9 inhibitors, lipoprotein apheresis, and even liver transplantation—often fall short in terms of efficacy, tolerability, or patient adherence. This creates a critical unmet need for durable, one-time therapies that can address the root cause of disease.

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.

To learn more, please visit: www.yoltx.com

SOURCE YolTech Therapeutics