- Orchard Therapeutics plc (NASDAQ:ORTX) has provided updates on the progress of its lead gene therapy programs targeting metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I Hurler syndrome (MPS-IH), and Wiskott-Aldrich syndrome (WAS).
- After receiving feedback from the FDA, the company expects to file a marketing application for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023.
- The submission of the marketing application in Europe for OTL-103 in WAS has been delayed to 2022 from 2021. The timeline on application 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.
- Price Action: ORTX shares are down 3.81% at $4.67 during the market session on the last check Tuesday.
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