Direct Lentiviral Injection 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 functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.

Timeline

Start

2025-05-31

Primary completion

2029-12-31

Completion

2030-12-31

Drugs

Indications