Gene

Genetic enhancement

Immunosuppression

Phase (matter)

Adverse effect

Biology

Adeno-associated virus

Surgery

Virology

Clinical trial

Biochemistry

Vector (molecular biology)

Genetics

Pediatrics

Organic chemistry

Internal medicine

Medicine

Chemistry

Capsid

Gastroenterology

Virus

Recombinant DNA

Factor IX

New England Journal of Medicine

FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B