OTL-203

OTL-203 is an investigational autologous ex vivo hematopoietic stem cell (HSC) gene therapy developed for the treatment of mucopolysaccharidosis type I (MPS I), specifically the Hurler subtype (MPS-IH). The therapy involves collecting a patient’s own CD34+ hematopoietic stem and progenitor cells, which are then genetically modified outside the body using a lentiviral vector to introduce a functional copy of the IDUA gene. This modification enables the cells to produce alpha-L-iduronidase, an enzyme deficient in MPS-IH patients. After modification, these cells are infused back into the patient intravenously. The goal is long-term metabolic correction by enabling continuous endogenous production of alpha-L-iduronidase, potentially addressing multiple disease manifestations not effectively treated by current standards such as allogeneic HSCT or enzyme replacement therapy[1][2][3][4][5][7].

Other names

autologous CD34+ hematopoietic stem and progenitor cells genetically modified with IDUA LV encoding alpha-L-iduronidase cDNA