Gene Therapy Program | FDA-Approved Gene Therapies

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 multiple FDA-approved gene therapy treatments, and we are actively participating in clinical trials in hopes of bringing additional approved gene therapies to our patients.

We are a designated qualified treatment center (QTC) for the following therapies:

• KEBILIDI™ gene therapy for AADC Deficiency
• CASGEVY™ and LYFGENIA™ gene therapy for sickle cell disease
• ELEVIDYS gene therapy for Duchenne muscular dystrophy
• KYMRIAH™ gene therapy for leukemia and lymphoma
• LUXTURNA™ gene therapy for inherited retinal disorder
• ROCTAVIAN™ gene therapy for hemophilia A
• SKYSONA® gene therapy for cerebral adrenoleukodystrophy (CALD)
• ZOLGENSMA® gene therapy for spinal muscular atrophy (SMA)
• ZYNTEGLO™ and CASGEVY™ gene therapies for beta thalassemia

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.

• More information on gene therapy for beta thalassemia.

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.

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.

ROCTAVIAN™ is designed for men 18 and older with severe hemophilia A without Factor VIII inhibitors. Given as a one-time intravenous infusion, it uses a viral vector to deliver the gene for clotting Factor VIII to reduce uncontrolled bleeding. It was approved in June 2023.

KYMRIAH™ is a form of gene therapy that alters the body's T-cells to better detect and destroy infected ‭or cancerous cells.‬‬‬‬‬‬ It is also the first FDA-approved CAR T-cell therapy.

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is offering KYMRIAH™ to two groups of eligible patients:

• Patients up to 25 years of age with relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL)
• Patients aged 18 and older with relapsed or refractory large B-cell lymphoma

Learn about CAR T-cell therapies for ALL and large B-cell lymphoma.

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.

• Learn about retinal gene therapy.

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.

• More information on gene therapy for sickle cell disease

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.

• Learn about SMA gene therapy.