Dr. Charles C. Wykoff discusses how a one-time gene therapy could benefit patients with wet age-related macular degeneration.
Although successful treatments are available to treat patients with wet age-related macular degeneration, new options — including one-time gene therapies — are needed, Charles C. Wykoff, M.D., Ph.D., said in an interview ahead of the American Society of Retina Specialists 2024 meeting in Stockholm. Wykoff is a board-certified Medical and Surgical Retina Specialist and ophthalmologist with Retina Consultants of Texas.
Wet AMD is an advanced form of the disease that can cause rapid and severe vision loss. About 20 million people in the United States have some form of AMD, and of those, about 1.5 million have late-stage AMD, which includes wet AMD.
Current treatment for wet AMD focus on endothelial growth factor (VEGF), which is believed to play a significant role in the abnormal formation of blood vessels in the macula. But they require repeat treatments, with some dosed every month with intravitreal injections, where medication is delivered to the back of the eye.
“The long term treatment burden is quite substantial for patients who receive shots in the eye repeatedly, often indefinitely,” Wykoff said in an interview before the meeting. “There’s a tremendous amount of data now showing that the consistency of appropriate treatment drops off over time.”
Gene therapies, however, have high price tags, often costing $2 million or $3 million or more. The most recently approved gene therapy, Lenmeldy, for juvenile metachromatic leukodystrophy, a life-threatening inherited disease of the body’s metabolic system, comes with a $4.25 million price tag.
“Our current therapeutics [for AMD] are also expensive,” Wykoff said. “My priority is figuring out what’s going to achieve the best outcomes in the safest way for patients.”
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