SBC-103 is an investigational enzyme replacement therapy for mucopolysaccharidosis IIIB (MPS IIIB), also known as Sanfilippo B syndrome. MPS IIIB is caused by a marked decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death. SBC-103 is the recombinant form of the human alpha-N-acetyl-glucosaminidase (NAGLU) enzyme.
Using various dosing approaches, SBC-103 demonstrated cellular uptake and reduced HSD substrate storage in the brains, liver and kidney tissues in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the U.S. Food and Drug Administration and the European Medicines Agency.
For more information regarding MPS IIIB, please visit the following patient organizations: