Abstract
The accessibility of the retina makes it an attractive target for genetic therapies to treat inherited retinal disorders (IRDs). The FDA's approval of voretigene neparvovec-rzyl in 2017 for biallelic RPE65-associated retinal dystrophies demonstrated that the safety and efficacy needed for regulatory approval of such therapies could be achieved. Clinical trials assessing a growing array of genetic strategies are offering valuable insight on the potential of these therapies for the treatment of IRDs while also emphasizing ongoing challenges in their development. This presentation will review the range of genetic therapies for IRDs being tested in clinical trials and the results to-date as well as discuss challenges including those related to evaluating efficacy.
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