Recently,Lingyi Biotech Co., Ltd.(hereinafter referred to as "Lingyi Biotech”）Announce,ItsLingyi Biotech's self-developed gene therapy drug for Wilson's disease, LY-M003 Injection, has officially received the implied permission for a Phase I clinical trial (IND) as a Class I therapeutic biological product from the National Medical Products Administration (NMPA).(Application No.: CXSL2600187)This is another key milestone achieved by Lingyi Biotech after the product received IND approval from the U.S. Food and Drug Administration (FDA) in November 2025 and was granted direct entry into Phase II clinical trials.About Hepatolenticular Degeneration
Hepatolenticular Degeneration(WD), also known as Wilson's disease, is a rare autosomal recessive genetic metabolic disorder. The disease is caused by mutations in the copper-transporting ATPase β (ATP7B) gene on chromosome 13, leading to the accumulation of copper ions in organs such as the liver, brain, and kidneys, causing organ damage. The clinical manifestations of WD are diverse, mainly including liver and nervous system lesions, such as abnormal liver function, enlargement of the liver or spleen, acute liver failure, neurodegeneration, etc.Current treatment methods include copper chelators and zinc agents, but they have many shortcomings, such as frequent medication, poor efficacy, and significant individual differences. As an emerging treatment method, gene therapy has the potential to fundamentally improve the symptoms of Wilson's disease with a single administration.LY-M003 Injection is a gene therapy drug independently developed by Lingyi Biotech for Wilson's disease. It is the world’s first AAV gene therapy that adopts dynamic copper ion regulation expression technology.Using recombinant adeno-associated virus (rAAV) as a vector, the therapy transfers the functionally normal ATP7B gene into patients' hepatocytes via a single intravenous infusion, enabling long-term stable expression of ATP7B protein with normal copper-transporting activity in hepatocytes. This innovative treatment aims to restore copper metabolism capacity in hepatocytes, reduce copper ion accumulation in the body, and reestablish ceruloplasmin activity, thereby systematically improving systemic symptoms to achieve the therapeutic goal for Wilson’s disease.Previously, "A prospective, single-center, open, 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" has been conducted at the First Affiliated Hospital of Zhejiang University School of Medicine. Several adult and adolescent patients with Wilson’s disease have been enrolled and dosed. Preliminary clinical observations have demonstrated good safety, with enrolled subjects showing good infusion tolerability, no serious adverse events (SAE) reported, and promising therapeutic effects.Currently,LY-M003 InjectionAlready obtainedFDA Orphan Drug(Orphan-drug designation, ODD) and Rare Pediatric Disease Designation (RPDD), further affirm its unique value in treating specific diseases.Lingyi BiotechFounded in February 2021, Lingyi Biotech is a globally leading company specializing in the development of innovative drugs for monogenic hereditary 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, Lingyi Biotech 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 disorders, the company aims to provide patients with one-time solutions under the highest quality standards.Simbay Park is located in the Waigaoqiao Bonded Area, the core of Shanghai's Pudong New Area Free Trade Zone. It is an innovative service-oriented characteristic industrial base under the Xingbei Group, focusing on the incubation, cultivation, and transformation of medical enterprises.The park relies on the unique "FTAs + bonded" outward-oriented economic regional advantages. By gathering the whole industry chain service resources of CRO+CDMO+CSO+EHS in biopharmaceuticals, government innovation and entrepreneurship support policies, and numerous well-known investment institutions, it provides enterprises with truly in-depth technical incubation in the medical industry, becoming a steward for the transformation of corporate technology achievements, and strives to create an international biopharmaceutical industry innovation and entrepreneurship ecosystem.
