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Putting regenerative therapies on the map

Eye institute publishes roadmap for taking audacious goals to clinical trials
October 23, 2023

Members of the National Eye Institute's Audacious Goals Initiative (AGI) published an outline of milestones to reach before commencing clinical trials of regenerative therapies for retinal disease.
"We are now at a point where it becomes feasible to draft the roadmap that would be used to translate AGI preclinical findings into clinical trials for people who have irreversibly lost vision from retinal and optic nerve disease," the authors wrote in a commentary published by the journal Med.1

Irreversible blindness often results from conditions that cause the loss of light-sensing photoreceptors and other retinal neurons, such as retinal ganglion cells, which carry signals from the retina to the brain. Examples include the blinding diseases glaucoma and age-related macular degeneration. Less common causes include injury and rare inherited diseases such as retinitis pigmentosa.
NEI launched the AGI in 2014 to foster research of regenerative therapies for eye disease. With AGI funding, researchers have advanced retinal imaging technology, discovered factors that affect neuronal regeneration, and characterized models for testing potential therapies. 

If preclinical findings can be translated to human use, it would revolutionize the lives of millions of people worldwide, according to the authors.  

The article outlines safety considerations; patient selection, including which disease to go after first; biomarkers and endpoints to measure efficacy; clinical controls; regulatory factors; and commercial viability. 
The report is a follow-on to the authors' report on progress in regenerative medicine research for vision.2 

  1. Levin LA, Chiang MF, Dyer MA, et al. Translational roadmap for regenerative therapies of eye disease. Med. Sep 8 2023;4(9):583-590. doi:10.1016/j.medj.2023.06.005
  2. Van Gelder RN, Chiang MF, Dyer MA, Greenwell TN, Levin LA, Wong RO, Svendsen CN. Regenerative and restorative medicine for eye disease. Nature Medicine. 2022/06/01 2022;28(6):1149-1156. doi:10.1038/s41591-022-01862-8

NEI leads the federal government’s efforts to eliminate vision loss and improve quality of life through vision research…driving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit  

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit 

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