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Nightstar gene therapy used in first US patient with X-linked retinitis pigmentosa
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A patient at Bascom Palmer Eye Institute has become the first in the United States to receive an innovative gene therapy treatment for X-linked retinitis pigmentosa as part of a Nightstar Therapeutics clinical trial, according to a press release.
Julio Adorno Nieves, 23, of Puerto Rico, is the 16th patient worldwide to receive the treatment. The treatment was delivered to his left eye in a 99-minute surgery by Janet L. Davis, MD, professor of ophthalmology and holder of the Leach Chair in Ophthalmology, and Ninel Gregori, MD, associate professor of clinical ophthalmology.
“Julio’s surgery went very well, as we were able to cover the entire central area of the retina where he still had some limited vision,” Davis said in the release.
Bryon Lam, MD, professor of ophthalmology and holder of the Robert Z. & Nancy J. Greene Chair in Ophthalmology, who is the principal investigator in the phase 1/2 trial, recommended the gene therapy procedure for Nieves.
“Introducing a functional copy of the RPGR gene using an engineered viral vector can correct the underlying cause and induce a long-lasting therapeutic effect,” Lam said of the treatment. “With gene therapy, some patients will have their central vision improve, while others may not. Hopefully, as Julio’s central vision improves, his night vision will get better as well.”
Nieves’ two brothers also have XLRP, along with multiple other family members. He is hoping the gene therapy can help them as well, according to the release.