Report on the National Eye Institute Audacious Goals Initiative: Photoreceptor Regeneration and Integration Workshop

The National Eye Institute (NEI) hosted a workshop on May 2, 2015, as part of the Audacious Goals Initiative (AGI) to foster a concerted effort to develop novel therapies for outer retinal diseases. The central goal of this initiative is to "demonstrate by 2025 the restoration of usable vision in humans through the regeneration of neurons and neural connections in the eye and visual system.'' More specifically, the AGI identified two neural retinal cell classes-ganglion cells and photoreceptors-as challenging, high impact targets for these efforts. A prior workshop and subsequent white paper provided a foundation to begin addressing issues regarding optic nerve regeneration, whereas the major objective of the May 2015 workshop was to review progress toward photoreceptor replacement and identify research gaps and barriers that are limiting advancement of the field. The present report summarizes that discussion and input, which was gathered from a panel of distinguished basic science and clinical investigators with diverse technical expertise and experience with different model systems. Four broad discussion categories were put forth during the workshop, each addressing a critical area of need in the pursuit of functional photoreceptor regeneration: (1) cell sources for photoreceptor regeneration, (2) cell delivery and/or integration, (3) outcome assessment, and (4) preclinical models and target patient populations. For each category, multiple challenges and opportunities for research discovery and tool production were identified and vetted. The present report summarizes the dialogue that took place and seeks to encourage continued interactions within the vision science community on this topic. It also serves as a guide for funding to support the pursuit of cell and circuit repair in diseases leading to photoreceptor degeneration.