Gene therapy for age-related macular degeneration (AMD) is an innovative approach aimed at addressing both wet and dry forms of the disease by delivering genetic material to retinal cells to modify or enhance their function. Here's an overview:

### Gene Therapy for Wet AMD

- Mechanism: Wet AMD is characterized by abnormal blood vessel growth due to overproduction of vascular endothelial growth factor (VEGF). Gene therapy uses vectors, such as adeno-associated viruses (AAV), to deliver genes that enable retinal cells to produce anti-VEGF proteins continuously. This reduces the need for frequent anti-VEGF injections[1][4][5].

- Examples: RGX-314 and ADVM-022 are gene therapy candidates that code for anti-VEGF proteins. These therapies aim for long-term efficacy with a single injection[4][5].

- Benefits: A "one-and-done" treatment approach could significantly reduce the burden of repeated injections and clinic visits[1][4].

### Gene Therapy for Dry AMD

- Mechanism: Dry AMD involves inflammation caused by overactivation of the complement system. Gene therapy introduces genes (e.g., GT005) that produce proteins to regulate this immune response, potentially slowing or halting retinal degeneration[3].

- Delivery: AAV vectors are used to transport therapeutic genes into retinal cells via subretinal injections, often requiring surgical procedures like vitrectomy[3].

### Challenges and Progress

- While gene therapy shows promise, it remains experimental, with ongoing clinical trials assessing safety and efficacy. Risks include surgical complications and immune responses[2][8].

This approach represents a significant advancement in AMD treatment, offering hope for more sustainable and less invasive solutions.

Sources

[1] A New Generation of Gene Therapies as the Future of Wet AMD ... https://pmc.ncbi.nlm.nih.gov/articles/PMC10888617/

[2] How is the landscape for treatments for macular disease changing? https://www.macularsociety.org/about/media/news/2024/june/how-is-the-landscape-for-treatments-for-macular-disease-changing/

[3] Gene Therapy for Age-Related Macular Degeneration https://www.ndcn.ox.ac.uk/research/clinical-ophthalmology-research-group/trials/amd-gene-therapy

[4] Gene Therapy for AMD - American Academy of Ophthalmology https://www.aao.org/eyenet/article/gene-therapy-for-amd

[5] Gene therapy for neovascular age-related macular degeneration https://bjo.bmj.com/content/105/2/151

[6] World's first gene therapy operation for common cause of sight loss ... https://www.ox.ac.uk/news/2019-02-19-world%E2%80%99s-first-gene-therapy-operation-common-cause-sight-loss-carried-out

[7] Gene therapy and macular degeneration https://www.mdfoundation.com.au/news/gene-therapy-and-macular-degeneration/

[8] Can gene therapy cure age-related macular degeneration (AMD)? https://www.topdoctors.co.uk/medical-articles/can-gene-therapy-cure-age-related-macular-degeneration-amd