ORTX - Orchard Therapeutics updates on gene therapy programs

metamorworks/iStock via Getty Images Orchard Therapeutics (ORTX) is trading marginally higher in the pre-market with a ~6.0% gain after the company provided updates on the progress of its lead gene therapy programs. The experimental treatments OTL-200, OTL-203, and OTL-103 target metachromatic leukodystrophy ((MLD)), mucopolysaccharidosis type I Hurler syndrome (MPS-IH), and Wiskott-Aldrich syndrome ((WAS)), respectively. After the feedback received from the FDA, the company expects to file a Biologics License Application ((BLA)) for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023. The submission of a Marketing Authorization Application ((MAA)) in Europe for OTL-103 in WAS has been delayed to 2022 from 2021, and the timeline on BLA submission to the FDA, previously expected in 2022, is not finalized yet. For OTL-203, Orchard anticipates the initiation of a study in 2022 targeting MPS-IH with a revised trial protocol incorporating the guidance given by the FDA and the European Medicines Agency ((EMA)). A

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Orchard Therapeutics updates on gene therapy programs