Target Disease:
Corneal
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

In Mucopolysaccharidosis
Type 1 (MPS1) patients

Comparison of a healthy cornea and stroma
versus MPS1 cornea and stroma

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,
and proteoglycan
aggregates

LEARN MORE ABOut GB-501 iN CORNEAL CLOUDING

Prevalence of Corneal
Clouding
Corneal Clouding
80-98%
of patients suffering
from MPS1

Corneal clouding affects over 80% of MPS1 patients (mainly children), and up to 98% of patients with the most severe form of MPS1. MPS1 is considered an ultra-rare disease affecting 1 in 100,000 people worldwide.


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.

The Pipeline

GB-501

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.

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.
Explore Our Pipeline