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Next steps for Orchard Therapeutics’ OTL-203 gene therapy

4 December 2023

Scientific laboratory

Orchard Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).

The Fast Track designation received is intended to expedite the development of promising medicines that address serious medical needs. Prior to this, the global gene therapy leader received Rare Pediatric Disease (RPD) and priority medicines (PRIME) designations from the FDA and European Medicines Agency for OTL-203.

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