Work

Gene therapy for hemophilia

Year: 2024

Type: article

Abstract: ConceptGene therapy with adeno-associated virus (AAV) vectors treats hemophilia A or B by delivering a functional F8 or F9 gene to hepatocytes.This single infusion enables endogenous production of factor VIII (FVIII) or FIX protein, reducing bleeding in patients (Figure ) Evolution Long-term clinical studies have provided insight into the efficacy, safety, and durability of AAV-mediated gene therapies for hemophilia A 1,2 and B. 3-5 Gene therapies have now been approved for hemophilia A (valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec and fidanacogene elaparvovec) in select regions. ApplicationThe approved gene therapies are associated with a significant reduction in bleeding tendency and increased FVIII or FIX activity levels, without the need for additional FVIII or FIX replacement therapies, in the vast majority of patients.3,6 Future steps Improvements in AAV technology, protein expression, and immunogenicity may make AAV-mediated gene therapies more efficient, longer lasting, and safer for people with hemophilia A or B.

Source: Blood Advances

DOI: 10.1182/bloodadvances.2024013864

Authors: Peter J. Lenting, Sylvia Fong

Institutions: Inserm, Université Paris-Saclay, Hemostase Inflammation Thrombosis, BioMarin (United States)

Language: English

Topic: Hemophilia Treatment and Research

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,000