Etuvetidigene autotemcel is an ex vivo genetically modified autologous CD34+ haematopoietic stem and progenitor cell gene therapy. The genetically modified cells are capable of expressing the functional WAS protein. Following administration, the genetically modified cells engraft and repopulate the haematopoietic compartment. These genetically modified cells containing the corrected WAS protein (WASP) differentiate and produce biologically active lymphoid and myeloid progenitors whose progeny express WAS protein.
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Document | Type | Information Source | |
|---|---|---|---|
| WASKYRA Suspension for solution for infusion | MPI, US: SPL/PLR | FDA, National Drug Code (US) | |
| WASKYRA Dispersion for infusion | MPI, EU: SmPC | European Medicines Agency (EU) |
Etuvetidigene autotemcel is an active ingredient of these brands:
Lithuania (LT)United States (US)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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