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Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [32] It was approved for medical use in the European Union in May 2019, [33] and in the United States in August 2022.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [1] [5] [2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
There are two approved forms of gene therapy for beta thalassemia. [96] [97] Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia which adds a healthy beta-globin gene to the HSCs. [98] It was approved for medical use in the United States in August 2022.
Beremagene geperpavec (Vyjuvek): treatment of wounds. [2] Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia [3] Brexucabtagene autoleucel (Tecartus): treatment for mantle cell lymphoma and acute lymphoblastic leukemia [4] [5] Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor peripheral artery ...
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. [5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [8]
The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. FDA clears first CRISPR ...
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