Exagamglogene autotemcel is a cell therapy consisting of autologous CD34+ HSPCs ex vivo edited by CRISPR/Cas9-technology. The highly specific guide RNA enables CRISPR/Cas9 to make a precise DNA double-strand break at the critical transcription factor binding site (GATA1) in the erythroid specific enhancer region of the BCL11A gene. As a result of the editing, GATA1 binding is irreversibly disrupted and BCL11A expression reduced. Reduced BCL11A expression results in an increase in γ-globin expression and foetal haemoglobin (HbF) protein production in erythroid cells, addressing the absent globin in transfusion-dependent β-thalassemia (TDT) and the aberrant globin in sickle cell disease (SCD), which are the underlying causes of disease.
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 |
|---|---|---|
| B06AX05 | 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 | |
|---|---|---|---|
| CASGEVY Dispersion for infusion | European Medicines Agency (EU) | MPI, EU: SmPC | |
| CASGEVY Suspension for infusion | FDA, National Drug Code (US) | MPI, US: SPL/PLR |
Exagamglogene autotemcel is an active ingredient of these brands:
United States (US)Croatia (HR)Lithuania (LT)United Kingdom (UK)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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