Lingyi Biotech Initiates Clinical Trial of AAV Gene Therapy LY-M003 for Adult Patients with Wilson Disease

Hepatolenticular Degeneration (WD), also known as Wilson's Disease, is a rare autosomal recessive genetic metabolic disorder caused by mutations in the copper-transporting ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3), leading to impaired copper transport. This results 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.

The standard treatment for Wilson's disease currently includes copper chelators like penicillamine, which increase urinary copper excretion, and zinc agents that reduce copper absorption. Although widely used, these treatments have several limitations, such as the need for frequent daily dosing, limited efficacy for neurological symptoms, and significant individual variability in patient response and tolerance. If a patient progresses to the decompensated cirrhosis stage, liver transplantation may also need to be considered. Gene therapy, as an emerging treatment modality, has the potential to fundamentally and sustainably alleviate 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 uses recombinant adeno-associated virus (rAAV) as a vector to deliver the functionally normal ATP7B gene into patients' hepatocytes via a single intravenous infusion, enabling long-term stable expression of the ATP7B protein with normal copper transport activity in hepatocytes. This innovative therapy aims to restore copper metabolism capacity in hepatocytes, reduce copper ion accumulation in the body, and restore ceruloplasmin activity, thereby systematically improving systemic symptoms to treat Wilson's disease. Currently, LY-M003 Injection has received FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD).

About Lingyi Biotech

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 gene therapy pipelines. With a commitment to 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 disorders, Lingyi Biotech aims to provide patients with one-time solutions according to the highest quality standards.

Lingyi Biotech's AAV gene therapy drug LY-M001 Injection for Gaucher disease under research obtained the IND approval for new drug clinical trials from NMPA and FDA in January 2024, and has officially launched the Phase I clinical registration trial. It has also received the FDA’s Orphan Drug Designation (ODD). Currently, LY-M001 Injection has completed dosing in several adult and pediatric patients with Gaucher disease, and preliminary data shows that LY-M001 Injection is...SafetyAndGood performance in terms of effectiveness.