SOBI-003

SOBI-003 is a chemically modified variant of recombinant human sulfamidase developed using Sobi's proprietary glycan modification technology. It is designed as an enzyme replacement therapy for mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a rare and severe metabolic disorder caused by mutations in the SGSH gene. These mutations result in deficient activity of the enzyme N-sulfoglucosamine sulfohydrolase, leading to accumulation of heparan sulfate and progressive neurodegeneration. SOBI-003 aims to reduce heparan sulfate storage in affected cells by replacing the missing or defective enzyme. The drug has demonstrated the ability to cross the blood-brain barrier and reduce heparan sulfate levels in cerebrospinal fluid, serum, and urine. It was granted orphan drug designation and Fast Track status by regulatory agencies but development was discontinued after Phase I/II trials[1][3][4][5][6][7].

Other names

Modified recombinant human sulfamidaseRecombinant-sulfamidase-Swedish-Orphan-Biovitrum