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Dr Charles Wykoff: Gene Therapy for Wet AMD Would Be a Tremendous Opportunity


Gene therapies like RGX-314 are looking to treat very common diseases in ophthalmology, which would represent a tremendous opportunity for chronic disease management, said Charles C. Wykoff, MD, PhD, of Retina Consultants of Texas and the Blanton Eye Institute at Houston Methodist Hospital.

While the first FDA-approved gene therapy was in ophthalmology, it was for a small patient population. Now, gene therapies like RGX-314 are looking to treat very common diseases, which would represent a tremendous opportunity for chronic disease management, explained Charles C. Wykoff, MD, PhD, director of research at Retina Consultants of Texas; chair of research, Retina Consultants of America; and deputy chair of ophthalmology for the Blanton Eye Institute, Houston Methodist Hospital.

Wykoff presented data on RGX-314, a gene therapy being developed to treat wet age-related macular degeneration, during the Angiogenesis, Exudation, and Degeneration 2023 meeting.


How are gene therapies changing ophthalmology? What is the importance of the gene therapy, RGX-314, to treat wet age-related macular degeneration?

It's an exciting time for gene therapy and ocular diseases. It's fascinating that ophthalmology was the first place where there was a gene therapy that was FDA approved for gene replacement with Luxturna. That was a major advance in our field many years ago.

Now, there are multiple—dozens, literally—of gene therapies in clinical trials for various ocular diseases. Many of those are looking at relatively isolate isolated diseases like subsets of the retinitis pigmentosa population, for example, but some of them are looking at very common diseases. And that's what we're talking about here. Gene therapy for a niche disease is very important and moves us forward. But a gene therapy for the most common cause of irreversible blindness in our country would be a tremendous step forward for the opportunity for management of this chronic disease.

We've continued to bend the curve as far as reducing the burden of blindness for this disease. I think this would be a big step forward. This is not the only gene therapy, looking at production of an anti-VEGF protein. There are others also. Others that are in important clinical trials. So, we're learning a lot as we go forward with this program and others. But this is the program that's further along to date and has very consistent data across multiple programs at this point. We look forward to continuing to obtain longer term data as well as seeing ultimately the data from the pivotal trials.

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