Development of a Novel Encapsulated Non-Viral Cell- Based, BBB-Penetrant Therapy for MPS I

Introduction

• MPS I is caused by a deficiency of the lysosomal enzyme a-L-iduronidase (IDUA) leading to GAG accumulation in multiple tissues and organs
• This accumulation results in a complex array of progressive, multi-systemic pathologies, including CNS manifestations
• Approved therapies include enzyme replacement therapy (ERT), with chaperone and gene therapies under investigation
• Treatment with approved ERT does not adequately address CNS manifestations