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Lentiviral Hematopoietic Stem Cell 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 transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.
Official title: Lentiviral Vector-modified Autologous Hematopoietic Stem Cells for Metachromatic Leukodystrophy (MLD)
Key Details
Gender
All
Age Range
1 Month - 50 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2025-06-01
Completion Date
2030-09-30
Last Updated
2025-07-01
Healthy Volunteers
No
Conditions
Interventions
Lentiviral TYF-ARSA correction of patient's autologous HSCs
Infusion of lentiviral TYF-ARSA modified autologous HSCs at 1\~10x10\^6 gene-modified cells per kg body weight.
Locations (1)
Shenzhen Geno-Immune Medical Institute
Shenzhen, Guangdong, China