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

The European Medicines Agency ((EMA)) has granted Priority Medicines ((PRIME)) designation to Orchard Therapeutics' (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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Orchard Therapeutics' gene therapy nabs accelerated review in Europe for neurometabolic disease