Mucopolysaccharidoses (MPS) are autosomal recessive lysosomal storage disorders and San Filippo Syndrome (MPS IIIA) is the most common of all MPS where 1 in 70,000 children and more than 5,500 patients are born with this disorder.

 

MPS IIIA is caused by a deficiency in the lysosomal enzyme, Sulfamidase (N-sulfoglucosamine sulfohydrolase, SGSH) that leads to a buildup of undegraded heparan sulfate in the brain of these children resulting in profound cognitive deterioration and behavioral disorders. Enzyme replacement therapy can restore lysosomal function, however it is limited by the inability of enzyme to cross the blood brain barrier and into the brain. Currently, no therapies exist for the treatment of MPS IIIA.

 

TEGA is developing enzyme replacement therapy using recombinant lysosomal enzyme and direct infusion delivery into the brain to address the significant unmet need in MPS IIIA. This therapeutic delivery approach is considered safe and well tolerated.