Lingyi Biotech Launches Clinical Trial of LY-M003, a Novel Gene Therapy for Wilson Disease

Recently, "A Prospective, Single-Center, Open-Label, Single-Arm, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult Patients with Wilson’s Disease" was launched at the First Affiliated Hospital of Zhejiang University School of Medicine (hereinafter referred to as "FAHZU"). The project is led by Professor Chao-Hui Yu, the leading figure in gastroenterology. LY-M003 Injection is an innovative gene therapy drug developed by Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech").

The Department of Gastroenterology at Zhejiang Hospital No. 1 is not only the earliest established and largest clinical diagnosis and treatment center for digestive system diseases in Zhejiang Province, but also, with its excellent professional standards and academic standing, has become a National Key Clinical Specialty, a National Training Base for Digestive Endoscopy Diagnosis and Treatment Technology, a Key Medical Discipline of Zhejiang Province, and the leading unit in Zhejiang Province’s digestive disease research field.

Professor Chaohui Yu currently serves as the Vice President of the First Affiliated Hospital of Zhejiang University School of Medicine and the academic leader in gastroenterology. He is a professor of internal medicine, chief physician, and doctoral supervisor at Zhejiang University. A leading talent in scientific and technological innovation under the National High-Level Talents Program, he has been recognized as a leading innovator by the Ministry of Science and Technology and as an outstanding talent under the New Century Excellent Talents Program by the Ministry of Education. Professor Yu has successively been selected for the second and first levels of Zhejiang Province's 151 Talent Project, receiving key funding, and is also recognized as a high-level innovative talent in health by Zhejiang Province. Professor Yu has long been engaged in the diagnosis and treatment of digestive system diseases, clinical teaching, and research work. He is committed to studying the mechanisms of metabolic liver diseases and developing new drugs, leading several national-level scientific research projects, including the National Key R&D Program, the National Natural Science Foundation, and sub-projects of the 973 Program.

About Hepatolenticular Degeneration

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 and 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 hepatic and neurological lesions, including abnormal liver function, hepatomegaly or splenomegaly, acute liver failure, neurodegeneration, neurological dysfunction, selective neural vulnerability, and executive dysfunction, among others. Involvement of other systems may also occur and could progressively worsen during the course of the disease.

The current standard treatment for hepatolenticular degeneration includes copper chelators like penicillamine, which increase urinary copper excretion, and zinc agents that reduce copper absorption. Although widely used, these treatments have many shortcomings, 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 decompensated cirrhosis, liver transplantation may also need to be considered. As an emerging therapeutic approach, gene therapy holds the potential to fundamentally and sustainably improve the symptoms of hepatolenticular degeneration with a single administration.

About LY-M003

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 through a single intravenous infusion, enabling long-term stable expression of ATP7B protein with normal copper-transporting 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 achieve the goal of treating 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 leading innovative drug development company for monogenic inherited diseases. The company boasts an internationally top-tier team in gene therapy discovery, research and development, and industrialization, committing to the R&D and industrialization of First-In-Class gene therapy pipelines. With a focus on source innovation, the company 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 diseases, it 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, currently under research, obtained IND approval from the NMPA and FDA in January 2024 and has officially entered Phase I clinical trials. It has also received Orphan Drug Designation (ODD) from the FDA. Currently, LY-M001 Injection has been administered to several adult and pediatric patients with Gaucher disease, and preliminary data indicate that LY-M001 Injection demonstrates good performance in both safety and efficacy.

Source of the article:Lingyi Biotech