Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

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.

Timeline

Start

2025-06-01

Primary completion

2025-06-01

Completion

2030-09-30

Drugs

Indications