The advent of factor replacement therapies has brought remarkable improvements in hemophilia care. However, bleeds, joint disease, inhibitor development and treatment burden remain significant problems for patients with hemophilia.1,2
Novel therapeutic agents aim to address unmet needs with the current treatments. These new treatments include bispecific antibodies that mimic the coagulation function of FVIII.1
Gene therapy for hemophilia is currently still in development and under investigation, and is neither available nor proved safe or effective.1
Gene therapy is a novel approach that is being developed for the treatment of hemophilia.1
Gene therapy involves the introduction, removal or change of the genetic material, specifically DNA or RNA, in the cells of a patient to treat a specific disease.3
A gene therapy currently being investigated for the treatment of hemophilia, called gene transfer, involves the addition of a functional copy of a gene into the target cells to produce a functional protein.3-5
Gene therapy for hemophilia is currently still in development and under investigation, and is neither available nor proved safe or effective.1
Unlike factor replacement therapies that aim to replace the missing or defective factor proteins, non-replacement therapies mimic the procoagulant activity of the clotting factors or enhance coagulation by inhibiting physiological anticoagulants.2
Some examples of non-replacement therapies include2:
DNA, deoxyribonucleic acid; FVIIa, factor VIIa; FVIII, factor VIII; FVIII, factor VIIIa; FIXa, factor IXa; FX, factor X; FXa, factor Xa; IgG4, immunoglobulin G4; RNA, ribonucleic acid; TF, tissue factor; TFPI, tissue factor pathway inhibitor.
An overview of hemophilia, including the different types of hemophilia, their causes, consequences and prevalence.
What is hemophilia?
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