First UK patient treated with CSL Behring’s gene therapy for Haemophilia B
Ella Day | June 20, 2025 | News story | Research and Development | CSL Behring, Haematology, NHS England, cell therapy, gene therapy, haemophilia B, prophylactic infusions
CSL Behring has announced that the first patient in the UK has received HEMGENIX (etranacogene dezaparvovec) through the NHS, marking a significant milestone in haemophilia B treatment and NHS access to gene therapies.
The treatment was administered at Guy’s and St Thomas’ NHS Foundation Trust in London to a patient from the North East of England. HEMGENIX is the first and only gene therapy for haemophilia B recommended by NICE via its Single Technology Appraisal and the first made available through a managed access agreement under the Innovative Medicines Fund.
“This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients,” said Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’. “It is also a testament to the advancement of cell and gene therapies in the UK.”
Etranacogene dezaparvovec is a one-time intravenous gene therapy that enables the body to produce its own factor IX protein, addressing the condition’s underlying cause. It offers a potential alternative to lifelong prophylactic infusions, which currently place a significant burden on patients.
Kate Burt, CEO of the Haemophilia Society, called the treatment a “milestone” for people living with haemophilia B. “Gene therapy will allow those eligible to expand their horizons and live life to the full, free from the restrictions of regular injections.”
CSL Behring will continue working with NHS England to ensure broader access to the treatment across the UK.
Ella Day
20/6/25
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