Alumna leads first gene therapy surgery to treat vision loss
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Richard and Loan Hill Department of Biomedical Engineering alumna Safa Rahmani, a pediatric retina specialist at Ann & Robert H. Lurie Children’s Hospital of Chicago, recently led a team in the first gene therapy approved in the U.S. to treat children and adults with a form of vision loss that can result in blindness, LUXTURNA®.
A 7-year-old girl from Chicago in August was the first patient at the hospital to undergo the surgical treatment.
The U.S. Food and Drug Administration approved LUXTURNA® as the first directly administered gene therapy in the U.S. as a treatment for patients with a hereditary form of retinal degeneration, biallelic RPE65 mutation-associated retinal dystrophy. It is a progressive condition that leads to severe vision impairment during early childhood and adolescence. Eventually, many patients with this inherited retinal dystrophy become completely blind. LUXTURNA® works by replacing the affected gene of the sick retinal cells with a “normal” version of the gene. The healthier cells can then function better, which allows the patient to have improved vision.
Treatment involves a surgical procedure in which a trained retinal surgeon injects the medication under the retina. The two-part surgery (one for each eye) is about two hours long and the patient can go home the same day.
Rahmani completed her bachelor’s and master’s degrees in biomedical engineering in 2004 and 2007, respectively. She is also the Chief Medical Officer of RetMap, Inc., a startup she and her UIC advisor, Associate Professor John Hetling, founded years ago, which recently launched its first FDA-cleared product, a sensor to help detect progressive eye disease.