Autologous Cd34+ Haematopoietic Stem And Progenitor Cells Genetically Modified With The Lentiviral Vector Idua Lv, Encoding For The Alpha-L-Iduronidase Cdna

• A study to investigate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared w… Ended
  2022-500306-17-00 Phase III 2 countries Orchard Therapeutics (Europe) Limited
• Gene therapy study with autologous hematopoietic stem cells for patients affected by MPS-IH Ended
  2024-514870-29-00 Phase I (FiH) 1 country Orchard Therapeutics (Europe) Limited

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