An overview of gene therapies in the pipeline for ocular disorders

L. Ferr Desideri, A. Vagge, M. Nicolò, C. E. Traverso

Research output: Contribution to journalReview articlepeer-review


Recently, ocular gene therapy has been proven to be a promising emerging treatment option for ocular disorders, considering the encouraging clinical outcomes and safety profile. In particular, ocular gene therapy has been introduced for the treatment of several inherited retinal diseases mostly by implementing an adeno-associated virus vector. In 2017, voretigene neparvovec was the first gene therapy approved by the U.S. Food and Drug Administration for the treatment RPE65-related Leber congenital amaurosis and retinitis pigmentosa. Moreover, gene therapy is being examined in several different nonhereditary diseases, including wet age-related macular degeneration, diabetic retinopathy and glaucoma. In this review article, we discuss the current approaches investigating the role of gene therapy for ocular disorders. Nonetheless, we will discuss its potential clinical benefit for patients and the reduction of the treatment burden due to the longer duration.

Original languageEnglish
Pages (from-to)383-390
Number of pages8
JournalDrugs of the Future
Issue number5
Publication statusPublished - May 2021


  • Gene therapy
  • Inherited retinal dystrophies
  • Luxturna
  • Macular degeneration
  • Ocular gene therapy
  • Voretigene neparvovec

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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