Inherited retinal dystrophies (IRDs) encompass a group of rare disorders associated with genetic defects that cause progressive retinal degeneration. Patients have severe, bilateral and irreversible vision loss beginning in early to mid-life. There are more than 200 gene defects associated with the most common IRDs. The first FDA-approved gene therapy, voretigene neparvovec (Luxturna; Spark Therapeutics), was approved for IRDs caused by biallelic mutations in RPE65 in adult or paediatric patients. The therapy was approved by the European Medicines Agency (EMA) in November 2018. These landmark decisions could further open the doors to gene therapies for ophthalmic diseases.

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