Lingyi Bio's Next-Gen Tunable Gene Therapies Shine in Late-Breaking Sessions at ASGCT 2025

Lingyi Biotech presented significant scientific and clinical advancements during the Late-Breaking session, delivering oral reports on the clinical research outcomes of its gene therapy drug for Wilson's disease (also known as hepatolenticular degeneration) and LY-M003 injection for Gaucher disease type I, with LY-M001 injection showcasing important scientific value and clinical significance.

LY-M003：Next-Generation Adjustable Gene Vector Technology Redefines Treatment Standards for Wilson's Disease

As the world's first AAV gene therapy utilizing copper ion dynamic regulation expression technology, LY-M003 achieves on-demand expression of therapeutic proteins from the underlying mechanism through its exclusive CREATE™ (Copper Responsive Engineering of ATP7B Expression) system. This dynamically coordinates the efficacy of gene therapy with the pathological severity in patients with hepatolenticular degeneration, better ensuring the long-term safety and effectiveness of the gene therapy.

Since September 2024, the seven subjects enrolled have shown good tolerance to the infusion, with no serious adverse events (SAE) reported and no generation of anti-ATP7B antibodies (ADA). Most patients have completely stopped standard drug therapy, and patients with neurological symptoms have shown significant improvement in neurologic functions such as writing ability and language function in the UWDRS score. The serum ceruloplasmin levels of the subjects have significantly increased, and some patients' urinary copper levels have returned to normal. The successful implementation of LY-M003 gene therapy marks a shift in the treatment model for hepatolenticular degeneration patients from "lifelong medication" to "functional cure."

LY-M001: Global Leading Clinical Advancements in Gaucher Disease Treatment

LY-M001 for Gaucher Disease (GD) expresses glucocerebrosidase (GCase) with higher protein stability and lower immunogenicity through an AAV vector.^LY), combined with a proprietary gene expression system, addresses the issues of traditional enzyme replacement therapy (ERT) for Gaucher's disease, such as the need for frequent injections and limited improvement in certain organ symptoms.

In Phase I/II and IIT clinical trials, LY-M001 has completed enrollment of 11 Gaucher patients, ranking first globally in clinical progress. In terms of safety, all patients tolerated the infusions well, with no treatment-related SAEs occurring and no anti-GCase antibodies detected.^LYAntibody (ADA) production. One week after a single infusion of LY-M001, GCase activity in the peripheral blood of subjects returned to normal levels, followed by a gradual decrease in the disease-specific biomarker Lyso-GL1. Efficacy indicators significantly improved after administration, including increased hemoglobin and platelet levels, reduced spleen and liver volumes, and improved quality of life with alleviation of symptoms such as abdominal distension and fatigue. All subjects resumed normal work or study. LY-M001 demonstrated positive intervention in the pathological progression of Gaucher's disease patients.

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Dr. Lin Qing, founder and CEO of Lingyi Biotech, stated: "The presentation of results at this year's ASGCT marks a significant milestone in the implementation of Lingyi Biotech’s 'original innovation + global synchronization' R&D strategy. The design of our gene therapy drugs not only overcomes the limitations of traditional gene expression regulation mechanisms but also demonstrates the therapeutic value of 'one-time dosing, long-term benefits' in clinical settings, aligning closely with the cutting-edge explorations of leading global pharmaceutical companies in the field of gene therapy. Both LY-M001 and LY-M003 have received FDA Orphan Drug Designation and Pediatric Rare Disease Qualification. Moving forward, the company will accelerate the development of its pipeline and establish a precision medicine ecosystem for genetic disorders."

Lingyi Biotech was founded in February 2021 and is a global leader in the development of innovative drugs for monogenic inherited diseases. The company boasts a world-class team in gene therapy discovery, research, development, and industrialization, focusing on the R&D and commercialization of First-in-Class (FIC) gene therapy pipelines. With a commitment to original innovation, the company concentrates on disease areas with urgent clinical needs such as metabolism, the central nervous system, and ophthalmology. Starting with key technologies like animal models of genetic diseases, Lingyi Biotech aims to provide patients with one-time solutions adhering to the highest quality standards.