Hemgenix

Product Specs	Description
Manufacturer Name	CSL Behring
Indications	HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes.
Contraindications	None known
Viral Safety Process
Product Half-Life	Factor IX activity levels of 39% (± 18.7), 41.5% (± 21.7), 36.9% (± 606 21.4) and 36.7 (± 19.0) of normal, respectively, at 6, 12, 18 and 24 months.
Manufacturing Method	HEMGENIX (etranacogene dezaparvovec-drlb) is an adeno-associated viral vector-based gene therapy for intravenous infusion after dilution. HEMGENIX is a non-replicating recombinant AAV5 containing a codon-optimized DNA sequence of the gain-of-function Padua variant of human Factor IX (variant R338L), under control of a liver-specific promotor 1 (LP1).
Storage Requirements/Shelf Life
Before Dilution	HEMGENIX is shipped at 2°C to 8°C (36°F to 46°F). Upon receipt, store HEMGENIX vials in a refrigerator at 2°C to 8°C (36°F to 46°F). Store in the original carton until use. Protect from light until time of dilution and administration. Do NOT FREEZE.
After Dilution	Once diluted, store in the infusion bag protected from light. Store diluted in the infusion bag at 15°C to 25°C (59°F to 77°F). Infuse the diluted product within 24 hours after the dose preparation.
Nominal Vial Size & Diluent Volume	Each vial contains an extractable volume of not less than 10 mL, containing 1 x 10¹³ gc/mL.
Route of Administration	Intravenous Administration