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: Active, no longer recruiting

Phase of Trial: Phase II

Latest Information Update: 20 May 2025

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At a glance

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.
27 Jan 2025 Planned End Date changed from 3 Apr 2028 to 31 Jan 2026.

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