iduronidase

TEGA Therapeutics is developing a recombinant alpha-L-iduronidase (IDUA) enzyme replacement therapy for the treatment of Mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome. MPS I is a rare lysosomal storage disorder caused by a deficiency in the IDUA enzyme, which is essential for the breakdown of glycosaminoglycans (GAGs) such as heparan sulfate and dermatan sulfate. The resulting accumulation of GAGs leads to progressive multi-organ dysfunction and severe neurological complications. TEGA's therapeutic approach involves replacing the deficient enzyme to restore normal lysosomal degradation of these substrates. To address the challenge of delivering enzymes across the blood-brain barrier, the company is exploring advanced delivery technologies, including conjugation with guanidinylated neomycin (GNeo) and direct intracerebroventricular administration, supported by funding from the National Institute of Neurological Disorders and Stroke.

Other names

IDUAalpha-L-iduronidaserecombinant alpha-L-iduronidase