Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2024 Dec 2;13(12):28.
doi: 10.1167/tvst.13.12.28.

Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs

Affiliations
Review

Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs

Jacque L Duncan et al. Transl Vis Sci Technol. .

Abstract

Inherited retinal degeneration (IRD) disease and age-related macular degeneration (AMD) are leading causes of irreversible vision loss and blindness. Although significant progress has advanced the field in the past 5 years, significant challenges remain. The current article reviews the accomplishments and research advances that have fueled the development of treatments for patients with IRD and AMD, including the first approved gene-augmentation treatment for RPE65-related retinal degeneration and complement inhibition therapies to slow progression of geographic atrophy (GA) in AMD. The article outlines opportunities to address gaps and unmet needs that should lead to additional progress toward the development of treatments for patients with IRDs and non-neovascular AMD in the future.

PubMed Disclaimer

Conflict of interest statement

Disclosure: J.L. Duncan, Ascidian (F), PYC Therapeutics (F), Janssen (F), SepulBio (F); A. Bowman, None; A. Laster, None; C. Gelfman, Visgenx (C), UC Berkeley Forum for Collaborative Research (C), Bluegen Therapeutics Foundation (C), FeliQS Corporation (C); D.G. Birch, Nacuity (C), Beacon (C, F), 4D (C, F), Novartis (C), Biogen (F), Ocugen (F), PYC (F), ONL (C), Bluerock (C), Aldeyra (C), SepulBio (C); S.E. Boye, Atsena Therapeutics (C, F, O, P); S.P. Daiger, 4D Molecular Therapeutics (C); L. del Priore, None; D.J. Zack, Perceive Biotherapeutics (C, O, P), Astellas (C), Vision-Care (C), Life Biosciences (C); J.T. Handa, Cirrus Therapeutics (C), Character Biosciences (C), Seeing Medicines (C)

References

    1. Liao DS, Grossi FV, Mehdi DE, et al. .. Complement C3 inhibitor pegcetacoplan for geographic atrophy secondary to age-related macular degeneration a randomized phase 2 trial. Ophthalmology. 2020; 127(2): 186–195. - PubMed
    1. [No authors listed]. Pegcetacoplan (Syfovre) for geographic atrophy in age-related macular degeneration. Méd Lett Drugs Ther. 2023; 65(1673): 49–50. - PubMed
    1. Khanani AM, Patel SS, Staurenghi G, et al. .. Efficacy and safety of avacincaptad pegol in patients with geographic atrophy (GATHER2): 12-month results from a randomised, double-masked, phase 3 trial. Lancet. 2023; 402(10411): 1449–1458. - PubMed
    1. Russell S, Bennett J, Wellman JA, et al. .. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017; 390(10097): 849–860. - PMC - PubMed
    1. Drag S, Dotiwala F, Upadhyay AK.. Gene therapy for retinal degenerative diseases: progress, challenges, and future directions. Invest Ophthalmol Vis Sci. 2023; 64(7): 39. - PMC - PubMed

Publication types

MeSH terms

Substances