Lingyi Biotech's Gene Therapy LY-M003 Receives FDA Rare Pediatric Disease Designation for Wilson’s Disease

Recently,Shanghai FT1 Life Science ParkResident EnterprisesLingyi BiotechIndependently DevelopedLY-M003 InjectionObtainU.S. Food and Drug AdministrationRare Pediatric Disease Designation (RPDD) granted by the FDA`, for treatment`Hepatolenticular Degeneration (Wilson’s disease, WD), also known as Wilson's disease。

RPDDIt is a qualification designation for pediatric rare disease drugs targeting diseases with fewer than 200,000 patients in the U.S. that severely endanger the lives of children under the age of 18. The RPDD and Priority Voucher Program, initiated by the FDA, recognizes the significant need for therapies for pediatric rare diseases and aims to encourage the development of new treatments for these serious or life-threatening conditions. Under this program, sponsors will be eligible to receive a Priority Review Voucher (PRV) after the approval of a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease. The PRV can be used to accelerate the review of any subsequent product marketing application by six months or sold to a third party.

Hepatolenticular Degeneration (WD), also known as Wilson's Disease, is a rare autosomal recessive metabolic disorder caused by mutations in the copper-transporting ATPase β (ATP7B) Gene mutations lead to impaired copper transport capacity, resulting in the accumulation of copper ions in multiple organs such as the liver, brain, and kidneys, causing organ damage. The clinical manifestations of WD are complex, primarily involving liver and nervous system disorders, including abnormal liver function, hepatomegaly or splenomegaly, acute liver failure, neurodegeneration, neurological dysfunction, selective neural vulnerability, and executive dysfunction, among others. Other systemic involvement may also occur and could progressively worsen during the course of the disease.

Figure Caption: Manifestations of Wilson's Disease¹

About LY-M003

LY-M003 Injection is an AAV gene therapy drug independently developed by Lingyi Biotech for Wilson's disease. Using recombinant adeno-associated virus (rAAV) as a vector, it delivers the normal ATP7B gene into patients' hepatocytes through a single intravenous infusion, enabling long-term stable expression of functionally active ATP7B protein in hepatocytes. This restores copper metabolism in hepatocytes, reduces accumulated copper ions in the body, reactivates ceruloplasmin, and systematically improves systemic symptoms, thereby achieving the therapeutic goal for hepatolenticular degeneration.

Lingyi Biotech was founded in February 2021 and is a global leading innovative drug development company for monogenic inherited diseases. The company has an internationally top-tier team in gene therapy discovery, research and development, and industrialization, committed to the R&D and industrialization of FIC gene therapy pipelines. With a focus on original innovation, the company targets 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, it aims to provide patients with one-time solutions under the highest quality standards.

Shanghai FT1 Life Science Park (Abbreviation"Simbay Park, StarNorth · Free Trade Zone No.1") is located in the Waigaoqiao Bonded Area, the core of Shanghai's Pudong New Area Free Trade Zone, and is an innovative service-oriented characteristic industrial park under the StarNorth Group that focuses on the incubation, cultivation, and transformation of medical enterprises.The park relies on the unique "FTAs + bonded" outward-oriented economic zone advantages, and provides truly in-depth technical incubation for the medical industry by gathering the entire industrial chain service resources of CRO+CDMO+CSO+EHS in the biopharmaceutical field, government innovation and entrepreneurship support policies, and numerous well-known investment institutions. It has become a steward for enterprises in the transformation of technological achievements, and is committed to building an international biomedicine industry innovation and entrepreneurship base.