Next-Generation Therapy for Inherited Blindness

Stargardt Disease

As a result of bi-allelic mutations in Abca4, the retinas of Stargardt patients accumulate toxic bisretinoid compounds. Accumulation of these toxic compounds triggers inappropriate activation of a component of the immune system called Complement leading to damaged retinas (progressively growing area of dead retina and associated blind spot).

Normal Vision

Normal Vision

 

Normal Eye

Normal Eye

Normal Retina

Stargardt Eye

Stargardt Eye

Progressively growing area of dead retina and associated blind spot

Stargardt Vision

Stargardt Vision

 

Published data demonstrate that inhibition of Complement is >90% effective in preventing retinal cell death in the mouse model of Stargardt disease.

Inhibition of Complement activation by Oak Bay Biosciences' molecule can potentially prevent blindness in Stargardt patients.

Oak Bay Biosciences' molecule has the potential to be a best-in-class therapy for Stargardt disease.