Lingyi Bio's LY-M003 Gene Therapy Receives FDA Clearance to Advance Directly into Phase II Clinical Trial for Wilson Disease

LY-M003 InjectionIt is a gene therapy drug independently developed by Lingyi Biotech for Wilson's disease, using recombinant adeno-associated virus (rAAV) as a vector and carrying the CREATE gene expression regulation system with independent intellectual property rights. It delivers normal function through a single intravenous infusion.ATP7BGene transfer into patients' hepatocytes, enabling long-term stable expression of the ATP7B protein with normal copper-transporting activity. This innovative therapy aims to restore hepatocyte copper metabolism, reduce copper ion accumulation in the body, and reactivate ceruloplasmin, thereby systematically improving systemic symptoms to achieve the treatment goal for Wilson's disease.

Previously, "A prospective, single-center, open-label, single-arm, single-dose clinical study evaluating 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 Obtained FDA Orphan Drug Designation(Orphan-drug designation, ODD) andPediatric Rare Disease Qualification(Rare Pediatric Disease Designation, RPDD), further affirming its unique value in treating specific diseases.