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Advanced Therapies to Treat Diseases of the Eye

Two colleagues in the lab discussing results after looking into a microscope.

Our goal is to transform the lives of millions of patients suffering from partial to total vision loss by developing a platform of life-changing regenerative therapies that restore sight.

Our mission: Develop best-in-class cell therapy for corneal endothelial dystrophies

Corneal endothelial disease is a sight-threatening and debilitating condition affecting millions of people throughout the world. Composed of a single layer of cells located on the posterior surface of the cornea, the endothelium serves both barrier and pump functions, which are essential to the maintenance of corneal clarity and optimal vision.

The cells of the corneal endothelium are incapable of regenerating. Once these cells die – due to diseases such as Fuchs dystrophy, surgical trauma, and/or congenital dystrophies that lead to endothelial cell loss and vision degradation – they are gone forever. Symptoms of corneal endothelial disease may include blurring, glare, discomfort, and, in some cases, severe pain.

Although topical therapy can relieve symptoms of early-stage disease, the only treatments for more severe corneal endothelial dysfunction are full- or partial-thickness corneal transplantation, referred to as penetrating (PK) or endothelial (DSAEK, DMEK) keratoplasty, respectively.

 Illustration showing labelled parts of the human eye.

Corneal endothelial disease progression

Simulating the patient’s perspective

  • Patient with 20/20 vision
  • Patient sees glares and halos at night
  • Contrast and brightness diminish
  • Vision becomes blurred and uneven
  • Objects are opaque, difficult to distinguish

Treatment options

While these EK methods are effective, they are complex procedures that require a supply of donor corneas in a 1:1 ratio (one healthy donor cornea to treat each diseased one). The global shortage of donor corneas cannot address this significant unmet demand.1

 Microcopic view of healthy endothelial cells

Healthy endothelial cells

 Microcopic view of damaged endothelial cells (noticeable dark spots)

Damaged endothelial cells

Reference

  1. Gain P, Jullienne R, He Z, et al. Global survey of corneal transplantation and eye banking. JAMA Ophthalmol. 2016;134(2):167-173.