voda schreef op 1 juli 2021 13:49:
Orchard Therapeutics updates on gene therapy programs
Jun. 29, 2021 7:53 AM ET Orchard Therapeutics plc (ORTX)By: Dulan Lokuwithana, SA News Editor1 Comment
Gene therapy concept. Medical technology. Medtech.
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Orchard Therapeutics (NASDAQ: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 conference call on update is scheduled for today at 8:00 a.m. ET.
In May, the company said a registrational trial for OTL-203 for MPS-IH was likely by year-end 2021.
Now read: Orchard Therapeutics draws a lower price target at Cantor on delayed milestones