An overwhelming majority of rare diseases are the result of a single-gene defect, making them a potential target in the hunt for treatment. The U.S. Food and Drug Administration (FDA) has approved just 4 gene therapies to date, but with more than 900 investigational new drug applications for ongoing clinical studies related to gene therapies in the works,1 the time for these therapeutics to take center stage is fast approaching. 

One of those 4 approved gene therapies is for the treatment of biallelic RPE65 mutation-associated retinal dystrophy—a rare eye disease that can cause…

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