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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients with Wilson Disease
NCTID
NCT06650319
(View at clinicaltrials.gov)
Description
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, rAAV8 as the carrier of gene therapy products. After a single intravenous infusion, ATP7B protein can be specifically transduced to the target organ liver and expressed in hepatocytes for a long time.
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Development Status
Active
Indication
Wilson Disease
Disease Ontology Term
DOID:893
Compound Name
LY-M003
Sponsor
Chaohui Yu
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
10
Results Posted
Not Available
Therapy Information
Target Gene/Variant
ATP7B
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transdution
Vector Type
AAV8
Editor Type
Dose 1
Undisclosed dose escalation, 3 levels
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Early phase1
Submit Date
2024-10-15
Completion Date
2030-03-30
Last Update
2025-02-13
Participation Criteria
Eligible Age
18 Years - 60 Years
Standard Ages
Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
Resources/Links
Resources/Links
No External Links Available.