Gene therapy

It is an exciting time in gene therapy for haemophilia in Australia.

Gene therapy for haemophilia aims to provide the body with a functioning version of the factor VIII (8) or factor IX (9) gene so that the body can produce enough clotting factor for the blood to clot normally.

Gene therapy has been registered for use in some countries. In Australia there are currently a number of advanced experimental clinical trials for gene therapy. Some Haemophilia Treatment Centres are participating in the trials. People who are interested can discuss this with their Haemophilia Treatment Centre as this is constantly changing.

Gene therapy for haemophilia

Gene therapy for haemophilia
Play Video about Gene therapy for haemophilia

This resource, Gene Therapy for Hemophilia is published by World Federation of Hemophilia (WFH) and has been linked here with permission. © 2023 World Federation of Hemophilia https://sdm.wfh.org/

Gene therapy is still a relatively new treatment for haemophilia and there is a lot of research in this area and new information becoming available. Initial results are promising, with a decrease in the number of bleeding episodes and the need for people to have prophylaxis treatment.

There are a lot of important considerations before undertaking gene therapy, for example, will you be suitable to have gene therapy, how long will the effect of treatment last, side effects, etc. All of these need to be discussed with your Haemophilia Treatment Centre.

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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...

 News & Stories

HFA Gene Therapy Snapshot Survey

HFA would like to understand what our community members want from their haemophilia treatments and your perspectives on gene therapy in particular.

 National Haemophilia Journal

Gene therapy webinar

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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

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

 National Haemophilia Journal

New gene therapy resource

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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

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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.

 National Haemophilia Journal

Gene therapy

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Gene therapy for haemophilia has been in advanced clinical trials for some years now and this Congress was a timely forum to discuss and evaluate it critically. ROBYN SHOEMARK Robyn Shoemark is Clinical Nurse Consultant...

 National Haemophilia Journal

Haemophilia gene therapy snapshot survey

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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

Novel haemophilia therapies

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Living in the new world of novel haemophilia therapies ROBYN SHOEMARK Robyn Shoemark is Clinical Nurse Consultant Haemophilia/Haematology at the Kids Factor Zone, The Children’s Hospital at Westmead, Sydney, NSW Discussion about the impact of...

 National Haemophilia Journal

World Hepatitis Day 2024

bleeding disordersgene therapyliver healthWorld Hepatitis Day

World Hepatitis Day 2024 reminds us It’s time for action – hepatitis C and liver health remain important issues for our community and acting now is vital.

More information

For more information on gene therapy in haemophilia, speak to your treating haemophilia specialist doctor or a gene therapy expert who is undertaking clinical trials in haemophilia.

Click here for contact details of your local specialist Haemophilia Treatment Centre.

Sources and reviewers

Important Note: This information was developed by Haemophilia Foundation Australia for education and information purposes only and does not replace advice from a treating health professional. Always see your health care provider for assessment and advice about your individual health before taking action or relying on published information. This information may be printed or photocopied for educational purposes.

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