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As gene therapies advance through clinical trials, more and more of these treatments are receiving approval from the U.S. Food and Drug Administration (FDA). Boston Children's Hospital now offers several FDA-approved gene therapy treatments, and we are actively participating in clinical trials in hopes of bringing additional approved gene therapies to our patients.

Current gene therapies we are offering include:

Beta thalassemia gene therapies (Zynteglo®, Casgevy™)

Caused by mutations in the beta-globin gene, beta thalassemia prevents people from producing enough beta-globin, a key hemoglobin component. Boston Children’s Hospital is now offering two FDA-approved therapies for patients with beta thalassemia who need regular transfusions of red blood cells: Zynteglo®, approved in August 2022 for children and adults, and Casgevy™, approved in January 2024 for patients aged 12 and older. Both treatments reduce the need for transfusions.

Cerebral adrenoleukodystrophy gene therapy (SKYSONA®)

Cerebral adrenoleukodystrophy (CALD) is a rare genetic condition that causes the buildup of very long chain fatty acids in the brain. Boston Children’s Hospital is now offering SKYSONA®, also called elivaldogene autotemce or eli-cel, to eligible boys with CALD who are not yet experiencing symptoms. Boston Children’s helped pioneer SKYSONA®, which was approved by the FDA in September 2022. It is designed to replace the defective or missing ABCD1 gene, which controls the production of an enzyme that our body normally uses to breaks down fatty acids.

Duchenne muscular dystrophy gene therapy (Elevidys)

Elevidys, a gene therapy given through a single IV injection, was approved in June 2023 for ambulatory 4- and 5-year-old children with Duchenne muscular dystrophy. It enables the body to make a protein containing key sections of dystrophin proteins that are defective in the muscles of children with DMD. Boston Children’s participated in the clinical trials of this treatment.

Hemophilia A gene therapy (Roctavian™)

Roctavian™, designed for adults with severe hemophilia A, uses a viral vector to deliver the gene for clotting Factor VIII to reduce uncontrolled bleeding. It was approved in June 2023.

Leukemia and lymphoma CAR T-cell therapy (KYMRIAH™)

CAR (chimeric antigen receptor) T-cell therapy is a promising treatment that alters the body's T-cells to better detect and destroy infected ‭or cancerous cells.‬‬‬‬‬‬

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is offering the first FDA-approved CAR T-cell therapy, called KYMRIAH™, to two groups of eligible patients:

Girl wearing bicycle helmet while standing on bike path

CAR T-cell therapy helps hockey fan ice her leukemia

Ava, a rising sixth grader, was one of the first patients to undergo CAR T-cell therapy for acute lymphoblastic leukemia (ALL) at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

Retinal gene therapy (Luxturna™)

The Department of Ophthalmology at Boston Children’s Hospital is a Certified Center of Excellence for Luxturna™, an FDA-approved gene therapy for the treatment of inherited retinal disorders in patients over 12 months of age with mutations in the RPE65 gene.

Sickle cell disease gene therapy (Casgevy™, Lyfgenia™)

Casgevy and Lyfgenia were both approved in December 2023. Casgevy uses CRISPR gene editing to silence a gene called BCL11A in red blood cells that normally shuts off fetal hemoglobin production, based on technology developed at Boston Children’s over decades. Lyfgenia is a lentiviral gene therapy that adds a healthy copy of the globin gene.

Spinal muscular atrophy (SMA) gene therapy (Zolgensma®)

Boston Children’s Hospital is one of the first pediatric hospitals in the nation to offer Zolgensma®, an FDA-approved gene therapy for the treatment of spinal muscular atrophy (SMA) in children less than 2 years of age.

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Sofie’s story: A new gene therapy treatment for SMA

One year after receiving gene therapy treatment through a clinical trial at Boston Children’s Hospital, Sofie is not only able to breathe on her own and eat by mouth, but is also sitting up, moving her head, raising her hands, saying words, and rolling in her small wheelchair.