Trial Profile

A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

Status: Completed

Phase of Trial: Phase II

Latest Information Update: 20 Mar 2026

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At a glance

16 Mar 2026 Status changed from active, no longer recruiting to completed.
13 May 2025 According to an Orchard Therapeutics media release, company published a manuscript (Long-term effects of atidarsagene autotemcel for metachromatic leukodystrophy) in the April 24 issue of New England Journal of Medicine.
13 May 2025 According to an Orchard Therapeutics media release, company will present data from this study at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting taking place May 13-17 in New Orleans.

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