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

• MPS I is caused by 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

HYPOTHESIS: therapeutic effect can be achieved with sustained levels of an hIDUA fusion enzyme capable of penetrating the BBB via administration of hIDUA fusion-secreting allogeneic human cells shielded within spheres designed to avoid immune rejection and pericapsular fibrotic overgrowth (PFO) in the patient