The treatment landscape for bleeding disorders is changing
It is an exciting time in gene and emerging therapies for bleeding disorders in Australia. The aim of the Gene & Emerging Therapies (GETs) hub is to give the bleeding disorders community up-to-date information and knowledge about all these different treatments and to help make a shared decision with family and your HTC as they become available.
Gene therapy
Other new and emerging treatments
Making decisions about treatment
Information for health professionals (coming soon)
Resources
Rebalancing therapies fact sheet
This factsheet explains how rebalancing therapies work in haemophilia and are given, types (TFPIs, siRNAs) and side effects.
Gene therapy booklet
Gene therapy for haemophilia Information for people with haemophilia about gene therapy and how it works in haemophilia. This booklet explains the types of gene therapy that are being trialled in haemophilia at present, who...
Gene therapy for haemophilia fact sheet
This fact sheet answers how gene therapy for haemophilia works, who can have it, the risks and benefits, the treatment process and other FAQs.
Steve on gene therapy for haemophilia
Steve shares his experience having gene therapy for severe haemophilia A – and how it has changed his life
Bispecific antibody therapy fact sheet
This factsheet on bispecific antibody therapies explains factor VIIIa (8a) mimetics like emicizumab, how they work and are given in haemophilia.
Haemophilia Treatment Preferences Survey
(HaemPref Survey) How do individuals make decisions about treatment? What factors encourage or discourage patients from treatments? What are individuals’ treatment goals? Results presented are based on a study conducted by Community and Patient Preference...
ISTH 2024 Congress: novel therapies
Exciting new haemophilia treatments using a range of innovative technologies were highlighted at ISTH 2024 Congress.
Gene and emerging therapies in haemophilia
Exciting advancements in gene therapy and other emerging treatments for haemophilia were presented at the Blood 2024 conference.
WFH Summit: new therapies and approaches
WFH CCS topics included health literacy in shared decision-making, new therapies for VWD and women and girls with bleeding disorders.
Prof Cedric Hermans on New Haemophilia Treatments and more
Prof Cedric Hermans spoke with HFA about new treatments for bleeding disorders and the importance of diagnosis for women and girls, and the broader community
Haemophilia gene therapy snapshot survey
HFA’s haemophilia gene therapy snapshot survey asked people and parents of children with haemophilia what outcomes they would like from treatment now and in the future.
From the President
As we wrap up 2024, HFA reflects on our achievements in new therapies, hep C advocacy and support during the year.
Gene therapy interview with Dr Michiel Coppens now available
On 14 May 2024 we were fortunate to have Dr Michiel Coppens join us at the HFA office for an interview about gene therapy. Watch it now.
New haemophilia therapies
It is an exciting time with more new haemophilia treatments likely to become available in Australia, but there have been a lot of questions about them and what they will mean for treatment and care....
Haemophilia treatment survey
SUZANNE O'CALLAGHAN Suzanne O'Callaghan is HFA Policy Research and Education Manager What information does our community want about new haemophilia treatments? And for those who are affected, what would you like haemophilia treatment to achieve...
Emerging therapies publications
Downloadable education resources on new and emerging therapies in bleeding disorders, such as gene therapy for haemophilia.
From the President
So much is happening and changing very quickly – hepatitis C, women and girls, the conference and new therapies.
New Therapies Webinar
Dr Liane Khoo (Haematologist and HTC Director, Royal Prince Alfred Hospital), Dr Sally Campbell (Haematologist, and acting HTC Director, Royal Brisbane & Women’s Hospital), Stephen Matthews (Clinical Nurse Consultant, HTC, Royal Prince Alfred Hospital) and Sharon...
New haemophilia therapies
ALEX KLEVER Alex Klever is Clinical Nurse Consultant – Haemophilia at the Queensland Haemophilia Centre, Royal Brisbane & Women’s Hospital Plenary – Treatment landscape into the future Chair ~ David FaganFrom then to now ~...
ISTH 2025 – emerging therapies in haemophilia and VWD
At ISTH 2025 researchers shared advances in emerging therapies for haemophilia and von Willebrand disease (VWD).
Acknowledgments
The Gene and Emerging Therapies Hub has been funded by education grants from CSL Behring and Sanofi. This funding is based on a mutual understanding that HFA will act independently for the benefit of the bleeding disorders community. HFA does not promote any particular pharmaceutical product. Where possible products are referred to by their class or generic name and any mention of any commercial brand is strictly for educational purposes.