SANFILIPPO Therapeutics

Sanfilippo Therapeutics is a biotechnology company dedicated to developing curative treatments for Sanfilippo syndrome, a devastating and rare neurodegenerative lysosomal storage disorder. The company focuses on advancing gene therapy candidates and small molecule approaches designed to address the underlying enzyme deficiencies in Mucopolysaccharidosis type III (MPS III). By leveraging specialized expertise in central nervous system delivery and lysosomal biology, Sanfilippo Therapeutics aims to halt disease progression and improve the quality of life for affected children. Their pipeline primarily targets the A and B subtypes of the syndrome, which are characterized by the toxic accumulation of heparan sulfate in the brain.