fidanacogene elaparvovec-dzkt (Beqvez)

Indications

• adenovirus-associated vector-based gene therapy indicated for treatment of adults with moderate to severe hemophilia B

Dosage

• 5 1011 vector genomes per kg
• IV infusion after dilution in 0.9%sodium chloride with 0.25% human serum albumin, final volume of 200 mL over ~60 minutes

Adverse effects

• hepatotoxicty
  • hepatocellular carcinoma?
• increased serum transaminases

More general terms

• gene therapy