This brand name is authorized in United States.
The drug WASKYRA contains one active pharmaceutical ingredient (API):
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UNII
SMN5E7TJ9C - ETUVETIDIGENE AUTOTEMCEL
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Etuvetidigene autotemcel adds full-length copies of the human Wiskott-Aldrich Syndrome (WAS) complementary deoxyribonucleic acid (cDNA) into patients' hematopoietic stem cells (HSCs) through transduction with WAS lentiviral vector (LVV). After infusion, the genetically modified cells engraft in the bone marrow, repopulate the hematopoietic compartment, and produce biologically active lymphoid and myeloid progenitors whose progeny express WAS protein (WASP). WASP regulates the structural protein actin in blood cells. |
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Below package inserts are available for further reading:
| Document | Type | Information Source | |
|---|---|---|---|
| WASKYRA Suspension for solution for infusion | MPI, US: SPL/PLR | FDA, National Drug Code (US) |
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