Development of a novel encapsulated non-viral cell-based therapy for MPS-6

Introduction

• Mucopolysaccharidosis type VI (MPS-6, Maroteaux-Lamy syndrome) is caused by a deficiency of the lysosomal enzyme arylsulfatase B (ARSB)
• ARSB deficiency results in incomplete or blocked degradation of glycosaminoglycans (GAGs), which accumulate in the lysosome and disrupt normal cell function
• Disruption of cell function manifests in symptoms of MPS-6:
• Short stature, coarse facial features, stiff joints, breathing problems, difficulty walking, hip pain
• Photo on the right shows rapidly progressing 16yr old male patient