Progress in Inherited Retinal Disease Drug Discovery and Development: A Foundation’s Perspective

Abstract

Ophthalmic drug discovery and development has enjoyed a recent renaissance, with a major shift away from reformulating old systemic drugs for ocular use to de novo discovery of drugs for specific ocular disease targets. This shift, coupled with a revolution in molecular biology and genetic sequencing, has uncovered an unprecedented number and variety of novel targets for therapeutic intervention in eye disease. With such a treasure chest of new science to pursue, it also creates a new challenge for translating the lab-based discoveries through the translational “valley of death” into full scale industry-led development of new, approved therapeutics to treat eye disease. This is in fact a daunting task, as the cost of drug development continues to increase and many of the new therapeutic targets are based on smaller, orphan diseases with very high unmet medical needs. This perspective focuses on the role of a nonprofit foundation, The Foundation Fighting Blindness, in fueling and supporting the advancement of new therapies for blinding inherited retinal degenerative diseases into approved therapeutics. The new collaborative model is changing the way breakthrough drugs are coming to market for patients, and innovative funding models are required to match the innovative science.