Haemevolution

This European website contains promotional content developed by CSL Behring for European healthcare professionals and includes references to CSL Behring medicines. Prescribing information and Adverse Event reporting can be found at the bottom of the page.

Haemevolution

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This European website, initiated and developed by CSL Behring, contains product specific information and promotional content on gene therapy in haemophilia B and is intended for an international audience of European healthcare professionals only. For more detailed information on the use of the product in your country, please visit www.cslbehring.com.

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*CSL Behring is legally obliged to restrict this website to healthcare professionals only. By clicking “Yes, I am a healthcare professional”, you confirm this statement is true and you accept all liability related. If you are not a healthcare professional you can visit our disease awareness webpage: www.haemevolution.eu

HEMGENIX® is the first and only approved gene therapy for haemophilia B in europe, addressing the root cause of haemophilia B and reducing its impact on patients.3

HEMGENIX® introduces a functional copy of the F9 gene to compensate for the F9 mutation. This pioneering gene therapy consists of a non-replicating, recombinant adeno-associated viral vector serotype 5 (AAV5), encoding the highly active FIX Padua variant protein under the control of a liver-specific promoter.1 This highly active FIX Padua protein variant generates 5-8 times higher mean endogenous FIX activity than the more common wild-type FIX protein.4,5

Superior Bleed Protection vs Routine FIX Prophylaxis1,2

READ MORE ABOUT HEMGENIX® EFFICACY

Eliminates the Need for Routine FIX Prophylaxis for the Majority of Patients1

READ MORE ABOUT HEMGENIX® EFFICACY

Elevated and Sustained FIX Activity For Years1,2

READ MORE ABOUT HEMGENIX® Durability

Safety Profile1,2

READ MORE ABOUT HEMGENIX® Safety
Gene Therapy in Haemophilia

Gene Therapy For Haemophilia Explained

Haemophilia A and B are both monogenic, X-linked genetic disorders that are suitable for gene therapy.2,6 Gene therapy for haemophilia A and B aims to increase clotting factor VIII and factor IX levels, respectively, by providing a new functional gene or coding sequence, leading to improved health outcomes, reducing - or possibly even temporarily eliminating - the need for routine factor prophylaxis.2,7,8

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Patient Experience

See the timeline of events experienced by haemophilia B patients on their road to HEMGENIX® treatment

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About HEMGENIX®

Discover more about HEMGENIX® design, development and Mechanism of Action.

References

  1. EU SmPC HEMGENIX® (European Medicine Agency, 2023)
  2. Pipe SW, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388:706-718.
  3. CSL Behring. First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission. Press release. 20 Feb 2023. Available at: https://newsroom.csl.com/2023-02-20-First-Gene-Therapy-for-Hemophilia-B,-CSLs-HEMGENIX-R-,-Approved-by-the-European-Commission. Accessed March 2023.
  4. Nathwani AC. Gene therapy for Haemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.
  5. Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis. 2021;2:1–14.
  6. Rodríguez-Merchán EC, et al. Gene Therapy in Hemophilia: Recent Advances. Int. J. Mol. Sci. 2021;22:7647-67.
  7. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for Haemophilia. Blood. 2019;133(5):407-414.
  8. Miesbach W, et al. Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B. Blood. 2018;131(9):1022-31.