Article Text

Download PDFPDF

Gene therapy for neovascular age-related macular degeneration: rationale, clinical trials and future directions
  1. Thales Antonio Cabral de Guimaraes1,2,
  2. Michalis Georgiou1,2,
  3. James W B Bainbridge1,2,
  4. Michel Michaelides1,2
  1. 1UCL Institute of Ophthalmology, University College London, London, UK
  2. 2Moorfields Eye Hospital, London, UK
  1. Correspondence to Michel Michaelides, Moorfields Eye Hospital, London EC1V 9EL, UK; michel.michaelides{at}


Age-related macular degeneration (AMD) is one of the leading causes of irreversible blindness in the developed world. Antivascular endothelial growth factor therapy has transformed the management and outcome of neovascular AMD (nAMD), although the need for repeated intravitreal injections—even lifelong—and the related complications, high drug costs, frequent clinic visits and repeated imaging have resulted in an enormous burden both to healthcare systems and patients. The application of gene therapy approaches for sustained delivery of a range of antiangiogenic proteins has the promise of helping to address these aforementioned challenges. A number of early phase clinical trials of gene therapy in nAMD have provided encouraging results, with many more ongoing or anticipated. There remain significant areas of controversy, including regarding the optimal treatment targets, routes of administration and potential safety concerns. In this review we aim to provide an update of the current status of gene therapy for nAMD and briefly discuss future prospects.

  • angiogenesis
  • clinical trial
  • degeneration
  • genetics
  • retina

This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See:

View Full Text

Statistics from


  • Funding This work has been supported by grants from the National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, Fight for Sight, Moorfields Eye Hospital Special Trustees, Moorfields Eye Charity, Retina UK, the Foundation Fighting Blindness (USA), and the Wellcome Trust (099173/Z/12/Z). The views expressed are those of the authors and not necessarily those of the NHS, the UCL, the NIHR or the Department of Health.

  • Competing interests MM, JWBB and MG consult for MeiraGTx.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; externally peer reviewed.

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.