Clinical Trial Initiation of Lingyi Biotech's LY-M003 Injection for Adult Wilson's Disease Patients

In September 2024, "A Prospective, Single-Center, Open-Label, Single-Arm Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Infusion of LY-M003 Injection in Adult Patients with Wilson's Disease" was initiated at the First Affiliated Hospital, Zhejiang University School of Medicine (FAHZU). The study is led by Professor Chaohui Yu, a leading expert in gastroenterology, as the Principal Investigator. LY-M003 injection is an investigational gene therapy drug developed by Lingyi Biotech Co., Ltd. ("Lingyi Biotech").

 

Clinical Trial Recruitment 

 

Dear Subject,

Zhejiang Hospital No. 1 is conducting a "A Prospective, Single-Center, Open-Label, Single-Arm Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Infusion of LY-M003 Injection in Adult Patients with Wilson's Disease" hereinafter referred to as "this study." This study has been approved by the Ethics Committee of Zhejiang Hospital No. 1 and plans to recruit approximately 5-10 adult patients with Wilson's disease at the Zhejiang Hospital No. 1.

If you or someone you know meets the following criteria, please contact us:

  1. Age ≥18 years and ≤60 years, with no gender restrictions.

  2. The subject fully understands the purpose, nature, and methods of the study, as well as possible adverse reactions, and volunteers as a subject, signing the Informed Consent Form voluntarily.

  3. Patients diagnosed with Wilson's Disease: Assessed with a Leipzig scoring system of ≥4 points.

  4. WD patients confirmed by laboratory testing to have double chromosomal mutations in the ATP7B gene.

  5. Subjects are either newly diagnosed with WD or have been treated for WD with standard therapy (such as D-penicillamine or zinc acetate) for at least 6 months prior to the screening period.

  6. Subjects have been restricting high-copper foods for at least 6 months prior to the screening period and agree to continue doing so during the study.

Your participation is entirely voluntary. If you are interested in participating, please contact the study physician, who will provide you with a detailed introduction to this study and the matters you need to cooperate with, and will preliminarily judge whether you meet the inclusion criteria. Even if you choose not to participate in this study, you will not be subject to any adverse effects, and your information will be kept confidential. Whether you can successfully participate in this study ultimately depends on the investigator's judgment. Your participation will contribute to the advancement of medical science, and we look forward to your involvement.

If you participate in this study, you will receive free drug treatment and related free examinations, as well as certain transportation and nutritional subsidies.

Contact: Clinical Coordinator

Contact Number: 19121572057

 

 

About FAHZU's Gastroenterology Department

FAHZU's Department of Gastroenterology is not only the earliest and largest clinical center for digestive diseases in Zhejiang Province but also a National Key Clinical Specialty, a National Training Base for Digestive Endoscopy, and the leading institution for digestive disease research in Zhejiang.

About Professor Chaohui Yu

Professor Chaohui Yu, Vice President of FAHZU and Academic Leader of Gastroenterology, is a Professor of Internal Medicine, Chief Physician, and Doctoral Supervisor at Zhejiang University. He has been recognized as a National Leading Talent in Science and Technology Innovation, a Ministry of Science and Technology Innovation Leader, and an Outstanding Talent of the Ministry of Education. His research focuses on metabolic liver diseases and novel drug development, and he has led multiple national research projects, including the National Key R&D Program and National Natural Science Foundation grants.

About Wilson's Disease (WD)

WD, also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by mutations in the ATP7B gene (13q14.3), leading to impaired copper transport and toxic copper accumulation in the liver, brain, kidneys, and other organs. Clinical manifestations include hepatic dysfunction, hepatosplenomegaly, acute liver failure, neurodegenerative symptoms, and neuropsychiatric impairments, which may progressively worsen.

Current treatments (copper chelators like D-penicillamine and zinc salts) require lifelong, frequent dosing, exhibit variable efficacy, and may cause significant side effects. In advanced cases, liver transplantation may be necessary. Gene therapy offers a potential one-time curative approach by addressing the root genetic defect.

About LY-M003 Injection

LY-M003 is an investigational AAV-based gene therapy designed to deliver a functional ATP7B gene via single intravenous infusion. The therapy enables long-term hepatic expression of functional ATP7B protein, restoring copper metabolism, reducing systemic copper overload, and normalizing ceruloplasmin activity to alleviate WD symptoms.

LY-M003 has received FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), highlighting its potential to address unmet medical needs in WD.