Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

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.

NCT Number	NCT07046338
Lead Sponsor	Shenzhen Geno-Immune Medical Institute
Conditions	Metachromatic Leukodystrophy (MLD)
Enrollment	10 participants
Start Date	2025-06-01
Primary Completion	2025-06-01 (estimated)
Study Completion	2030-09-30 (estimated)
Updated on ClinicalTrials.gov	2025-07-01