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FDA-Approved Gene Therapies

FDA-approved gene therapies at Boston Children's Hospital

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.

Boston Children’s Hospital is a designated Qualified Treatment Center (QTC) for the following FDA-approved gene therapies:

FDA-approved gene therapies for beta thalassemia in children and adults

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.

FDA-approved gene therapy for cerebral adrenoleukodystrophy (CALD) in boys

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.

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Gene therapy for adrenoleukodystrophy: Risks, benefits detailed

Two studies report exciting long-term results of gene therapy for cerebral adrenoleukodystrophy (CALD), but also highlight a risk for cancer.

Read more

FDA-approved gene therapy for Duchenne muscular dystrophy in children

ELEVIDYS is a one-time intravenous gene therapy approved for boys and men aged 4 and older with genetically confirmed Duchenne muscular dystrophy (DMD). It delivers a gene that enables the body to produce micro-dystrophin, a shortened but functional form of dystrophin that helps slow disease progression. Boston Children’s Hospital participated in the clinical trials of this therapy.

FDA-approved gene therapy for hemophilia A in adult men

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.

FDA-approved CAR T-cell therapy for leukemia and lymphoma for young adults

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:

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

Read Ava's story

FDA-approved gene therapy for metachromatic leukodystrophy in children

LENMELDY™ is a one-time gene therapy for children with early-onset metachromatic leukodystrophy (MLD), a rare metabolic disease. It uses a child’s own stem cells modified to carry a functional ARSA gene to help break down harmful substances and slow disease progression. It was approved by the FDA in 2024.

FDA-approved gene therapy for inherited retinal disorders in children

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.

FDA-approved gene therapies for sickle cell disease in children and young adults

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.

FDA-approved gene therapy for spinal muscular atrophy (SMA) in babies and toddlers

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.

Read Sofie's story

FDA-approved gene therapy for aromatic L-amino acid decarboxylase deficiency in children

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that affects the brain’s ability to produce essential neurotransmitters like dopamine. Boston Children offers KEBILIDI™ (eladocagene exuparvovec-tneq) to eligible children with AADC deficiency. It is the first gene therapy for AADC deficiency to be approved by the FDA, and the first approved gene therapy to be delivered directly into the brain. KEBILIDI™ introduces a functional copy of the DDC gene into specific areas of the brain to enable production of the AADC enzyme. Clinical studies have shown improvements in motor function — including head control, ability to sit, and in some cases, walking — especially in younger patients.

Meet the experts behind our Gene Therapy Program

Our world-renowned team of pediatric gene therapy specialists is here to help your child access the most advanced FDA-approved treatments.

Meet our gene therapy team

Contact Boston Children’s Gene Therapy Program

Whether you're seeking care, a second opinion, or help navigating insurance coverage, our expert team in the Gene Therapy Program is here to guide you every step of the way.