Therapy for Stargardt Disease and Geographic Atrophy
BackgroundStargardt disease affects approximately 30,000 people in the United States, and geographic atrophy affects approximately 1 million people. These progressive retinal degenerative disorders are believed to be caused by the accumulation of toxic vitamin A dimer in the retina, leading to photoreceptor loss.
Our ApproachbiOOrg3.14 developed an oral small molecule—a chemically modified vitamin A—designed to reduce toxic vitamin A dimer formation in the retina.
Potential ImpactIf effective, this therapy could preserve vision for significant patient populations, reduce the economic burden of retinal degeneration, and address a leading cause of untreatable blindness.
StatusLicensed to Alkeus Pharmaceuticals.
Late-stage clinical development, with Phase 3 trials underway.