Dosing & Uses
Hemophilia B
Adeno-associated virus vector-based gene therapy pending FDA approval for hemophilia B
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Pharmacology
Mechanism of Action
Vaccine contains a bioengineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX (FIX) gene
The gene therapy is to enable treated individuals to produce FIX themselves via this one-time treatment rather than needing regular IV infusions of FIX, as is the current standard of care
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
