Lingyi Bio's First-in-Class Tunable AAV Gene Therapy LY-M003 Granted FDA Clearance to Proceed Directly to Phase II Clinical Trial for Wilson Disease

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On November 18, 2025, Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech" or "the Company") reached another key milestone in its globalization journey:Lingyi Biotech's self-developed gene therapy drug LY-M003 Injection for the treatment of Wilson's disease has received the Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) and has been authorized to directly enter Phase II clinical trials.This milestone marks an important step for Lingyi Biotech in its expansion into the international market, not only validating the potential efficacy and safety of LY-M003 but also indicating a significant acceleration in the international development of its core pipeline.

LY-M003 is the world's first AAV gene therapy employing dynamic copper ion regulation technology.Lingyi Biotech owns the full independent intellectual property rights of the CREATE™ system. This system can dynamically adjust the expression level of the ATP7B protein based on the copper metabolism condition in a patient’s body, ensuring dynamic coordination between the gene therapy effect and the severity of the pathological condition in the patient's body, thereby enhancing the safety and efficacy of the treatment.

At the beginning of this month, the latest research results of LY-M003 injection were first announced at the 76th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, The Liver Meeting 2025), the world's top academic conference on liver diseases, drawing significant attention from peer experts in the field. From its impressive debut at the academic forefront to the smooth passage through regulatory review, Lingyi Biotech has successfully achieved a key transition from "showcasing potential" to "delivering value." This not only demonstrates its strong independent R&D capabilities but also highlights the comprehensive strength and determined progress of Chinese innovative pharmaceutical companies in addressing unmet clinical needs on a global scale.

Previously, a clinical study titled "A Prospective, Single-Center, Open-Label, Single-Arm, Single-Dose Study to Evaluate the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult and Pediatric Patients with Wilson’s Disease" was initiated at the First Affiliated Hospital of Zhejiang University School of Medicine. The study has completed enrollment and dosing of 7 adult patients and the world’s first adolescent patient with Wilson’s disease. Preliminary clinical observations have demonstrated good safety, with enrolled subjects showing good infusion tolerability, no serious adverse events (SAEs) reported, and promising therapeutic effects.

Currently, LY-M003 injection has received FDA Orphan-drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), further affirming its unique value in treating specific diseases.

Source: Lingyi Biotech