WELCOME TO THE

Gene & Emerging Therapies Hub

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.

What's new?

Resources

Filter by content type (taxonomy) - no title
  • Articles
  • News
  • Personal stories
  • Videos
  • Fact sheets & booklets
  • FAQs
Fact sheet / Booklet

Gene therapy

AAV vectorsclinical trialsgene therapyhaemophilia

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...

 National Haemophilia Journal

Gene and emerging therapies in haemophilia

emerging therapiesgene therapyhaemophiliatreatment

Exciting advancements in gene therapy and other emerging treatments for haemophilia were presented at the Blood 2024 conference.

 National Haemophilia Journal

ISTH 2024 Congress: novel therapies

emerging therapieshaemophiliatreatment

Exciting new haemophilia treatments using a range of innovative technologies were highlighted at ISTH 2024 Congress.

 National Haemophilia Journal

WFH Summit: new therapies and approaches

emerging therapiesgirlsshared decision-makingVWDWFH CCSWomen

WFH CCS topics included health literacy in shared decision-making, new therapies for VWD and women and girls with bleeding disorders.

 National Haemophilia Journal

Haemophilia gene therapy snapshot survey

gene therapyhaemophiliaoutcomestreatment

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.

 National Haemophilia Journal

Haemophilia Treatment Preferences Survey

emerging therapiesgene therapyhaemophiliaoutcomesresearch studiestreatment

(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...

 National Haemophilia Journal

Jack’s latest travel adventures

adventure sporthaemophiliatravelYouth

Jack talks about his travel adventures in Canada and the USA, snowboarding and managing haemophilia on the road with new treatments.

 National Haemophilia Journal

Haemophilia treatment survey

EHLsgene therapyhaemophiliaoutcome measuressurveystreatment

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...

Information

Gene therapy FAQs

Is gene therapy a cure for haemophilia? In many cases successful gene therapy means that a person with severe haemophilia now has mild haemophilia, although a small number now have normal factor levels. It is...

 National Haemophilia Journal

From the President

Conference 2025emerging therapiesgirlsHepatitis CrepresentationWomen

So much is happening and changing very quickly – hepatitis C, women and girls, the conference and new therapies.

 Video

New Therapies Webinar

gene therapy

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...

 National Haemophilia Journal

New haemophilia therapies

gene therapyhaemophilianon-factor therapy

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 ~...

 Video

Gene Therapy Interview with Dr Michiel Coppens

gene therapy

In this interview chaired by Dr Stephanie P’ng and led by questions from the community, Dr Michiel Coppens explains what gene therapy is, how it works, and how it can be used to treat people...

 National Haemophilia Journal

Gene therapy webinar

gene therapyhaemophilia

Dr Michiel Coppens joined us at HFA for an interview about gene therapy, answering questions from the community. Watch the video or read the transcript.

 National Haemophilia Journal

Congress – a Foundation perspective

gene therapytreatmentWFH World CongressWomen

Haemophilia Foundation Australia President Gavin Finkelstein spoke with HFA about his experiences at the WFH 2024 World Congress.

 News & Stories

Haemophilia – the spice of life

careermusictransition

Willem spoke to us about working, exercising and being out and about in these days with new treatments.

 National Haemophilia Journal

New gene therapy resource

gene therapyhaemophilia

HFA’s new resource Gene therapy for haemophilia was developed to answer the questions from our community:   What types of gene therapy are used in haemophilia? How does gene therapy work? Is it a cure?...

 National Haemophilia Journal

From the President

Bleeding Disorders Awareness Monthgene therapyhaemophiliarepresentation

Heading to the end of the year, we review our progress. Gene and emerging therapies are on the agenda, and Bleeding Disorders Awareness Month is coming up.

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.

Join the HFA community

Sign up for the latest news, events and our free National Haemophilia magazine

Skip to content