MPS IIIA

Sanfilippo A (mucopolysaccharidosis IIIA; MPS IIIA) is a rare genetic lysosomal storage disorder (LSD) affecting about 1 in 150,000 births, with prevalence as high as 1/24,000 in some regions.  MPS IIIA is caused by a genetic defect in the gene for the lysosomal enzyme heparan N-sulfatase (N-sulfoglucosamine sulfohydrolase; SGSH) and is characterized by relatively mild somatic features but severe neurological manifestations (decline of learning abilities, hyperactivity, behavior problems, sleep difficulties, seizures) leading to dementia and death during puberty or early adulthood.  

Our goal is to develop an effective enzyme replacement therapy (ERT) for Sanfilippo A patients by exploiting the safety, supply, and cost advantages of plant-based enzyme bioproduction and integrating novel enzyme delivery strategies from our recent research program.