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).
Progressively growing area of dead retina and associated blind spot
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.