YolTech Therapeutics Reports 24-Week Clinical Data for YOLT-101 in Nature Medicine

Single dose of YOLT-101 led to durable LDL-C reductions >50% at Week 24

YOLT-101 was well tolerated with a favorable and manageable safety profile

SHANGHAI – March 4, 2026 – YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene-editing therapies, today announced the publication of 24-week clinical data for YOLT-101 in Nature Medicine.

The peer-reviewed article, entitled “In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial,” reports results from an ongoing first-in-human, single-ascending dose early clinical study evaluating YOLT-101 in adults with heterozygous familial hypercholesterolemia (HeFH).

YOLT-101 is an in vivo base editing medicine that uses LNP to encapsulate hpABE5 (a novel high-precision adenine base editor developed by YolTech) mRNA and guide RNA targeting the PCSK9 gene.

In the study, six participants received a single intravenous infusion of YOLT-101 across three dose levels (0.2 mg/kg, 0.4 mg/kg, and 0.6 mg/kg).

At the highest dose (0.6 mg/kg, n=3), YOLT-101 reduced circulating PCSK9 by 74.4% at Week 24 and lowered LDL-C by 52.3% at Week 24, with reductions of LDL-C evident as early as at Week 4 (48.9%). Lipid lowering was dose-dependent and remained durable through 24 weeks following a single administration.

YOLT-101 was generally well tolerated across all dose cohorts. No serious adverse events, Grade ≥3 adverse events, treatment-related discontinuations, or deaths were reported. Transient infusion-related reactions were primarily observed at higher dose levels and resolved within 24 hours. Reversible, asymptomatic elevations in liver enzymes were observed in three participants and were not associated with bilirubin increases.

The publication also includes extensive preclinical genome-wide analyses demonstrating no detectable off-target editing within assay sensitivity limits, supporting the high precision profile of the hpABE5 adenine base editor.

The full publication is available at: https://www.nature.com/articles/s41591-026-04254-4

About YolTech

Built on HEPDONE™ Novel Editor Platform and non-viral LNP technologies, YolTech Therapeutics is advancing in vivo gene-editing medicines with the potential for a one-time treatment that provides lifelong benefit. The company’s expanding clinical pipeline addresses genetic, metabolic, cardiovascular, and autoimmune diseases, with early clinical results supporting the potential for durable and transformative outcomes.