Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …

Background Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy
vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX …

Gene therapy has garnered increasing interest over recent decades. Several therapies
employing gene transfer mechanisms have been developed, and, of these, adeno …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …

Purpose To report historical patterns of pharmaceutical expenditures, to identify factors that
may influence future spending, and to predict growth in drug spending in 2023 in the United …

Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general
standard of care for severe hemophilia is frequent intravenous recombinant or plasma …

Adeno-associated viral vector (AAV) gene therapy provides a promising platform for
treatment of monogenic inherited disorders. Clinical studies have demonstrated long-term …

Gene transfer therapies utilizing adeno-associated virus (AAV) vectors involve a complex
drug design with multiple components that may impact immunogenicity. Valoctocogene …