azamidugene autotemcel

Azamidugene autotemcel (OTL-203) is an investigational ex vivo autologous hematopoietic stem and progenitor cell (HSPC) gene therapy developed for the treatment of Mucopolysaccharidosis type I Hurler (MPS-IH). The therapy consists of autologous CD34+ cells obtained from mobilized peripheral blood, which are genetically modified ex vivo using a self-inactivating lentiviral vector to express a functional copy of the human alpha-L-iduronidase (IDUA) gene under the control of a human phosphoglycerate kinase (PGK) promoter. Following a myeloablative conditioning regimen, the transduced cells are reinfused into the patient, where they engraft in the bone marrow and differentiate into various blood cell lineages. These cells express and secrete supraphysiologic levels of the functional IDUA enzyme, which is taken up by bystander cells via mannose-6-phosphate receptors, leading to systemic and central nervous system clearance of accumulated glycosaminoglycans (GAGs).

Other names

IDUA-encoding lentiviral vector transduced autologous hematopoietic stem and progenitor cellsautologous CD34+ cells transduced with lentiviral vector encoding human alpha-L-iduronidase