iduronicrin genleukocel-T

A preparation of autologous plasmablasts genetically engineered to express alpha-L-iduronidase (IDUA; laronidase) using the Sleeping Beauty (SB) transposon system, that may potentially be used for enzyme replacement therapy for Mucopolysaccharidosis type I (MPS I; Hurler syndrome). Upon administration, iduronicrin genleukocel-T expresses and replaces IDUA. IDUA, an enzyme required for the lysosomal degradation of glycosaminoglycans (GAGs), hydrolyzes the non-reducing terminal alpha-L-iduronic acid residues in GAGs, including dermatan sulfate and heparan sulfate. IDUA deficiency causes accumulations of heparan sulfate and dermatan sulfate in various tissues of the body which leads to progressive damage. Mutations in the IDUA gene are responsible for the deficiency of IDUA.