Lifelong VISION

In Lifelong VISION, we will combat vision loss by developing effective therapeutic approaches for IRD and MD, and by matching patient characteristics with their optimal treatment(s) at the right time. Blindness is considered the worst possible health outcome by at least half of the world’s population. Two devastating eye disorders are disrupting the lives of the young and of the old: inherited retinal dystrophies (IRD) and age-related macular degeneration (AMD). In the last decade, enormous efforts have been made to disentangle disease pathomechanisms and develop revolutionary therapies such as gene therapy and regenerative medicine, but these advances are still far out of reach for most patients. There is a clear need for innovations in conceptual and computational technology that enables us to shift into a higher gear and develop therapies that are customized to the cause, disease stage, and patient profile. Such technology is instrumental to achieve lifelong vision for all. Lifelong VISION will generate a pipeline of therapies applicable to the entire trajectory of retinal degeneration in five domains: Pillar 1 will develop and demonstrate proof-of-concept of gene-based therapies targeting DNA or RNA for permanent or transient repair of the genetic defect. Pillar 2 will develop and demonstrate proof-of-concept of pathway-based therapies targeting prominent disease pathways which cause retinal degeneration. Pillar 3 will develop and demonstrate proof-of-concept of cell-based therapies replacing lost retinal cells. Platform A will deliver model systems that closely mimic the back of the eye. Platform B will develop data-driven patient profiles tailored to each intervention modus and a core outcome set for clinical trials.