Gene Therapy for Neovascular Age-Related Macular Degeneration: Rationale, Clinical Trials and Future Directions

Age-related Macular Degeneration (AMD) is the leading cause of irreversible blindness in the elderly. The use of anti-angiogenic therapy has revolutionised the management and outcomes of neovascular AMD (nAMD). However, the need for repeated intravitreal injections, in some cases lifelong, and the related complications, frequent visits, elevated drug costs and repeated imaging, have resulted in an unsustainable burden both to patients and healthcare systems. Gene therapy aims to achieve a sustained delivery of anti-angiogenic factors, with the promise of addressing these challenges. Several nAMD gene therapy trials—both in early and late stages—have been completed and provided results which are encouraging, with many more trials currently underway or anticipated in the near future. Some areas of debate remain, such as routes of administration, targets for optimal treatment and safety concerns. This chapter aims to provide an update of the current situation of gene therapy for nAMD and discuss future prospects.