Betibeglogene autotemcel
| Clinical data | |
|---|---|
| Trade names | Zynteglo |
| Other names | LentiGlobin BB305, autologous CD34+ cells encoding βA-T87Q-globin gene |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a622065 |
| License data |
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| Pregnancy category | |
| Routes of administration | Intravenous |
| ATC code | |
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Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
The most common adverse reactions include reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch).
It was approved for medical use in the European Union in May 2019, and in the United States in August 2022.