Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

NARecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

June 1, 2025

Primary Completion Date

June 1, 2025

Study Completion Date

September 30, 2030

Conditions

Metachromatic Leukodystrophy (MLD)

Interventions

GENETIC

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.

Trial Locations (1)

RECRUITING

Shenzhen Geno-Immune Medical Institute, Shenzhen

All Listed Sponsors

lead

Shenzhen Geno-Immune Medical Institute

OTHER

NCT07046338 - Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD | Biotech Hunter | Biotech Hunter