LTE001

LTE001 is an enzyme replacement therapy (ERT) being developed by Letolab for the treatment of Mucopolysaccharidosis type I (MPS I). MPS I is a rare lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase, which is essential for the degradation of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate. The accumulation of these GAGs leads to progressive multi-organ dysfunction. LTE001 provides a recombinant version of the alpha-L-iduronidase enzyme to replace the deficient endogenous enzyme, thereby facilitating the breakdown of accumulated GAGs and mitigating the clinical manifestations of the disease. The program is currently in the IND-enabling stage of development.