Target Disease:Corneal
Clouding
in MPS1
Patients
Clouding
in MPS1
Patients
MPS1 is a rare, autosomal-recessive lysosomal storage disease caused by a deficiency of alpha-L-iduronidase (IDUA), an enzyme that breaks down intracellular and extracellular glycosaminoglycans (GAGs). In the absence of functional IDUA, the accumulation of GAGs results in enlarged cells and tissues throughout the body, manifesting as a progressive, life-threatening, multi-system disease. In its most severe form, MPS1 results in fatality in late childhood.
Corneal Clouding
Prevalence of Corneal
Clouding
Clouding
Corneal Clouding
80-98%
of patients suffering
from MPS1
from MPS1
The Unmet Need of
Patients with CORNEAL CLOUDING
The approved treatments for MPS1, such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplants (HSCT), have greatly increased life expectancy of MPS1 patients.
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.
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.
The Pipeline
GB-501
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.


While enzyme replacement therapy (ERT) and hematopoietic stem cell transplants (HSCT) have greatly increased life expectancy, these systemic treatments are unable to reach the cornea to prevent clouding, and the resulting loss of vision impacts more than 98% of MPS1 patients.