Gene Therapy Program | FDA-Approved Gene Therapies (2024)

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.

Gene Therapy Program | FDA-Approved Gene Therapies (2024)
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