New gene therapy resource

Gene therapy for haemophilia
Do you have questions about gene therapy for haemophilia?

We are pleased to announce the release of HFA’s new resource Gene therapy for haemophilia, which 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?
  • How safe is it?
  • Who can have gene therapy?

The information includes diagrams to explain simply how genes and gene therapy work.

gene therapy infusion process


Gene therapy for haemophilia is available in multiple formats:


We would like to thank the bleeding disorders community members and expert reviewers who made valuable contributions to this resource, and to our designer, Ray Hehr, for his clear and thoughtful diagrams.

In December 2018-January 2019 HFA conducted a national community survey to find out what questions the bleeding disorders community would like to have answered. This resource was developed to answer those questions.

This resource was funded by an education grant from BioMarin.


To find out more about Gene therapy for haemophilia or to order copies, contact HFA:

T: 1800 807 173

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