Betibeglogene autotemcel adds functional copies of a modified β-globin gene into the patients' HSCs through transduction of autologous CD34+ cells with BB305 LVV, thereby addressing the underlying genetic cause of the disease. After betibeglogene autotemcel infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate to produce RBCs containing biologically active βA-T87Q-globin (a modified β-globin protein) that will combine with α-globin to produce functional Hb containing βA-T87Q-globin (HbAT87Q).
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC code | Group title | Classification |
|---|---|---|
| B06AX02 | B Blood and blood forming organs → B06 Other hematological agents → B06A Other hematological agents → B06AX Other hematological agents | |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Title | Information Source | Document Type | |
|---|---|---|---|
| ZYNTEGLO Dispersion for infusion | European Medicines Agency (EU) | MPI, EU: SmPC |
Betibeglogene autotemcel is an active ingredient of these brands:
Austria (AT)Cyprus (CY)Estonia (EE)France (FR)Ireland (IE)Italy (IT)Lithuania (LT)Poland (PL)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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