Advances in Gene Therapy for Retinal Diseases

Author(s)/Faculty: Byron L. Lam, MD; Ninel Gregori, MD; Mark Pennesi, MD, PhD, FARVO; Charles C. Wykoff, MD, PhD
Release Date: 10/31/2022Expiration Date: 10/30/2023
Credit Type: CMENumber of Credits: 1.5
Content Type: ArticleProvider:
Although there have been no additional approvals since voritegene neparvovec for inherited retinal disease, clinical research into gene therapy for both inherited and non-inherited retinal diseases have continued to progress, and several drug candidates are advancing through the development process. While some potential therapies have failed to demonstrate efficacy, others are showing promise as future effective treatment options. Accumulating data for some of these agents show good long-term outcomes following a single dose. Delivering these treatments is challenging, as subretinal administration is complex and invasive, while intravitreal therapy may be less effective and lead to inflammation. Suprachoroidal administration may prove to be a safe and effective alternative route. While clinical trials of gene therapy in non-inherited retinal diseases have produced some very positive outcomes, adverse events are possible and retinal specialists must be knowledgeable about these events, as well as how to manage them. Recent developments in the field have advanced understanding of the mechanisms responsible for vision loss, creating new opportunities to intervene in the course of disease by developing new therapeutic approaches. In this educational activity, expert faculty will review the rationale for the use of gene therapy and most recent clinical data supporting this therapeutic approach in retinal disease.