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Direct Lentiviral Injection Gene Therapy for MLD
Sponsor: Shenzhen Geno-Immune Medical Institute
Summary
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.
Official title: Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)
Key Details
Gender
All
Age Range
1 Month - 50 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2025-05-31
Completion Date
2030-12-31
Last Updated
2025-06-26
Healthy Volunteers
No
Conditions
Interventions
Intrathecal and intravenous LV gene therapy
Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 transduction units/ml which carry a normal ARSA gene
Locations (1)
Lung-Ji Chang
Shenzhen, Guangdong, China