Gene therapy for haemophilia
A fact sheet on gene therapy for people with haemophilia, their families and carers.
It explains what gene therapy is, the types of gene therapy in haemophilia and how they work; who is eligible for gene therapy; benefits, risks and side-effects; what the treatment process is and how your Haemophilia Treatment Centre can help with advice and support. It also answers FAQs. Includes a diagram on how gene therapy works and is given.
(October 2025)
Acknowledgments
This fact sheet has been funded by unrestricted 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.