Type 1 (MPS1) patients
Normal cornea is completely transparent
MPS 1 cornea becomes opaque
Normal corneal stroma is
transparent with organized
fibrils and normal cells
MPS 1 corneal stroma has
abnormal collagen fibrils,
vacuolated stromal cells,
The Unmet Need of
Patients with CORNEAL CLOUDING
However, these systemic treatments are unable to reach the cornea to prevent its clouding, and the resulting loss of vision. Currently, no cure exists to address MPS1 corneal clouding, which affects up to 98% of patients.
GB-501 is an AAV gene therapy with orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to treat corneal clouding in children suffering from Mucopolysaccharidosis Type 1 (MPS1), a rare lysosomal storage disorder.
GB-501 uses adeno-associated virus (AAV) as a ‘vector’ to deliver the functional IDUA gene directly to the stromal layer of the cornea of both early and advanced MPS1 patients.
Restoration of corneal clarity following single intrastromal injection
Clear cornea (with view on retina) post GB-501 injection
Clouded cornea control
Preclinical studies in a canine model of MPS1 demonstrated complete and sustained clearing of the cornea in all dogs, regardless of disease severity, in less than a month following a single intrastromal injection. Two-year animal data support the potential for GB-501 to cure corneal clouding and restore vision in MPS1 patients.
GB-501 is currently in preclinical development.