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Novel Therapieshemophilia_logo.pngNovel TherapiesEventshemophilia_logo.pngAboutAboutWhat is Hemophilia?Hemophilia Burden of DiseaseHeritageDiagnosisDiagnosisSigns and SymptomsInvestigationsTreatmentTreatmentTreatment OptionsNovel TherapiesSupport & ResourcesSupport & ResourcesPatient Support ResourcesHemMobile®Patient Support LeafletsEvents

Novel Therapies

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.
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Gene therapy for hemophilia is currently still in development and under investigation, and is neither available nor proved safe or effective.

Gene Therapy

 

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

Non-replacement Therapies

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
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Some examples of non-replacement therapies include2:

  • Subcutaneous bispecific monoclonal antibodies (IgG4) that mimic activated FVIIIa by bridging FIXa and the FX zymogen to enhance FX activation
  • Antibodies that bind to and inhibit the activity of TFPI. The TFPI brakes the initiation phase of coagulation by inactivating FXa and the FVIIa-TF complex. Because the binding of FXa enables subsequent inhibition of the FVIIa-TF complex, the TFPI operates in a negative feedback loop that regulates the generation of FXa. Blocking TFPI activity extends the initiation phase of the coagulation, which generates more FXa. Anti-TFPI products are currently still in development and under investigation, and are neither available nor proved safe or effective.
  • A subcutaneous double-stranded small interfering RNA that targets antithrombin to improve sufficient thrombin generation and promote hemostasis. Antithrombin small interfering RNA products are currently still in development and under investigation, and are neither available nor proved safe or effective.

 

   

  

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. 

  

References:Kizilocak H, Young G. Diagnosis and treatment of hemophilia. Clin Adv Hematol Oncol. 2019;17(6):344-351.Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. A molecular revolution in the treatment of hemophilia. Mol Ther. 2020;28(4):997-1015.Gene therapy basics. American Society of Gene & Cell Therapy. Accessed April 27, 2022. https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basicsGene and cell therapy FAQs. American Society of Gene & Cell Therapy. Accessed April 27, 2022. https://asgct.org/education/more-resources/gene-and-cell-therapy-faqsFuture therapies: frequently asked questions. National Hemophilia Foundation. Accessed April 27, 2022. https://www.hemophilia.org/bleeding-disorders-a-z/treatment/future-therapies/frequently-asked-questions#1

  

For the use only of Registered Medical Practitioners or a Hospital or a Laboratory.

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Novel Therapies         
About

An overview of hemophilia, including the different types of hemophilia, their causes, consequences and prevalence.
 

What is hemophilia?

   
Diagnosis

Refresh your knowledge about the symptoms and diagnosis of hemophilia and understand why early diagnosis and treatment may be important for your patients.

Signs and symptoms

   
Treatment

Understand the current treatment options and recommendations for patients with hemophilia A and B.
 

Treatment options

Adverse events should be reported. Reporting forms and information can be found at [URL]


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