recombinant sulfamidase

Recombinant sulfamidase is an enzyme replacement therapy (ERT) being developed by TEGA Therapeutics for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. MPS IIIA is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme N-sulfoglucosamine sulfohydrolase (SGSH), which is essential for the degradation of the glycosaminoglycan heparan sulfate. This deficiency leads to the toxic accumulation of partially degraded heparan sulfate within lysosomes, primarily affecting the central nervous system and resulting in progressive neurodegeneration and cognitive decline. TEGA Therapeutics' recombinant enzyme aims to restore lysosomal degradation activity. To bypass the blood-brain barrier, which typically limits the effectiveness of systemic ERT in treating neurological symptoms, the company is investigating direct-to-brain delivery methods, including intranasal and intracerebroventricular administration.

Other names

recombinant N-sulfoglucosamine sulfohydrolaserecombinant SGSHrecombinant human sulfamidaserecombinant human SGSHrecombinant human N-sulfoglucosamine sulfohydrolase