IT and IV Lentiviral Gene Therapy for X-ALD

Summary

This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.

Timeline

Start

2025-08-31

Primary completion

2027-12-31

Completion

2028-12-31

Drugs

Indications