MERIT White Paper:

Overview of Gene Therapy in Inherited Retinal Diseases

Inherited Retinal Diseases (IRDs) are a group of diseases that can cause severe vision loss or even blindness. Each IRD is caused by at least one gene that is not working as it should. IRDs are rare but can affect individuals of all ages and can progress at different rates. Clinical indications of IRDs include color or night blindness, peripheral visual defects, and subsequent vision loss.


Over 30 clinical trials in IRDs were underway in 2021, with more in the planning stages. These trials are exploring AAV vector gene therapy, CRISPR-Cas9 gene editing tools, anti-sense oligonucleotides, and stem cell replacement therapy.


Gene therapy has made important advances in the treatment of rare IRDs, offering hope to millions of people in preventing or reducing vision loss due to previously untreatable diseases. As the field continues to advance, important discoveries will be made regarding new vectors, delivery approaches, and innovative endpoints.

Learn about disease states, viral and non-viral vectors, routes of delivery, specialized endpoints, and future directions for gene therapy in the treatment of IRDs.


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