Orchard Therapeutics' gene therapy nabs accelerated review in Europe for neurometabolic disease

Sep. 28, 2020 7:19 AM ETOrchard Therapeutics plc (ORTX)ORTXBy: Vandana Singh, SA News Editor4 Comments
  • The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to Orchard Therapeutics' (NASDAQ:ORTX) OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).
  • MPS-I is a rare neurometabolic disease caused by a deficiency of the alpha-L-iduronidase lysosomal enzyme required to break down sugar molecules.
  • Recently, OTL-203 received US Orphan Drug designation for the indication.

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