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News — The Division of Ophthalmology at Ann & Robert H. Lurie Children’s Hospital of Chicago recently became an Ocular Gene Therapy Treatment Center for LUXTURNA® (voretigene neparvovec-rzl), the first gene therapy to treat children and adult patients with a form of vision loss that can result in blindness. Lurie Children’s became the first center in Illinois and the 15th center in the country to become a surgical site for LUXTURNA®.  

In August, a 7-year-old girl from Chicago became the first patient at Lurie Children’s to undergo the surgical treatment, which was administered by Lurie Children’s pediatric retina specialist Dr. Safa Rahmani.  She was assisted by Dr. Alice Lyon (Northwestern Medicine) and a team of pediatric eye experts including Drs. Jennifer Rossen and Brenda Bohnsack, the Head of Lurie Children’s Division of Ophthalmology.

“This therapy offers a lot of hope for children with RPE 65 retinal disease, and allows Lurie Children’s to become a center for future clinical trials with gene therapy,” said Dr. Rahmani. 

The U.S. Food & Drug Administration (FDA) has approved LUXTURNA® as the first directly administered gene therapy in the US. It is 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. This treatment allows Lurie Children’s to become a center for future eye gene therapy trials and treatments. 

For more information, or to make an appointment with one of Lurie Children’s hereditary retinal disease specialists for an evaluation, please call the Division of Ophthalmology, 312.227.6728.  

Lurie Children’s Division of Ophthalmology provides world-class treatment and care for a full range of pediatric eye disorders, eye trauma and vision problems. The division consists of ten board-certified ophthalmologists with fellowship training in pediatric ophthalmology and strabismus, and three optometrists with interests in pediatric care and low vision. The team also includes a pediatric retina specialist. Last year, the division offered care and consultations to approximately 28,536 patients and performed 1,324 surgeries.