ISTH 2024 Congress catch up on the latest data and insights into gene therapy for haemophilia B

In June, the International Society on Thrombosis and Haemostasis (ISTH) 2024 Congress took place in Bangkok, Thailand, where CSL Behring sponsored the symposium 'Building momentum to raise the standard of care in bleeding disorders' and product theatre 'Learning through shared experience: Gene therapy for haemophilia B'.

The expert faculty of the educational symposium, David Lillicrap (Canada), Ampaiwan Chuansumrit (Thailand), Pratima Chowdary (UK) and Robert Klamroth (Germany), took the audience on a journey exploring innovations in the treatment and diagnosis pathways of bleeding disorders, including haemophilia B and von Willebrand disease, where they explored how small steps can result in big leaps for patient care.

In the product theatre, the esteemed faculty Steven Pipe (USA), Doris Quon (USA) and Daan Breederveld (The Netherlands) led an exciting discussion around their experiences of the haemophilia B gene therapy journey, including an up-to-date overview of the current treatment landscape and exploring several different experiences shared from the clinician's and patient's perspective.

If you were unable to attend, or would like to watch either of the presentations again, they are available on-demand:

• Building momentum to raise the standard of care in bleeding disorders
• Learning through shared experience: Gene therapy for Haemophilia B

In the oral communication and poster networking sessions, new data were presented from clinical trials, which investigated etranacogene dezaparvovec gene therapy in patients with haemophilia B.

• The abstract 'Etranacogene dezaparvovec hemophilia B gene therapy phase 2b trial final results: stable and durable FIX level expression over 5 years' reported long-term data and demonstrated stable factor IX expression that is maintained for five years after a single infusion of etranacogene dezaparvovec.
• The abstract 'IX-tend: A phase IV observational, long-term follow-up study on the safety and efficacy of etranacogene dezaparvovec in patients with hemophilia B' reported the study design of this Phase 4, observational, post-authorisation, long-term follow-up, multicentre, international study.
• The abstract 'Validation of a neutralizing antibody (NAb) assay with extended reportable range for etranacogene dezaparvovec, an adeno-associated virus serotype 5 (AAV5)-based gene therapy for adult hemophilia B' reported validation data for a modified assay to extend the reportable measuring range of AAV NAbs, as patients with pre-existing AAV NAbs in this increased range may benefit from etranacogene dezaparvovec treatment.

Data from other CSL Behring-funded studies were also presented on haemophilia B prophylaxis therapy. If you would like to read any of the published abstracts from the ISTH Congress, they are available here.