First NHS patient receives gene therapy for haemophilia B

Written by Jessica Bomford, June 19, 2025

A 44-year-old man has become the first person to receive gene therapy for haemophilia B on the NHS.

The man, who is treated in Newcastle, travelled to London to receive the treatment, called which was administered by Dr Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’ Hospital, on 18 June.

first gene therapy administered for haemophilia B on 18 June 2025 at Guy's

As you can see from the picture, this potentially life-changing therapy, called Hemgenix, is administered in a single infusion which took about an hour. There will now be twice-weekly follow-ups checking liver function and factor levels.

He will hope to reach near-normal factor levels which should last for the foreseeable future without any further treatment. As gene therapy is a new treatment, this data is based on clinical trials which have taken place over the last 10 years. There may be large difference between how individuals respond to the therapy.

The first dosing of gene therapy for severe haemophilia B marks a landmark moment and we expect to see more people treated within the next few months at the eight designated centres across England.

Kate Burt, chief executive of the Haemophilia Society, said: ‘This marks an important milestone not just for this patient, but for all those living with haemophilia B in the UK.

‘The current treatment of lifelong intravenous injections can place a significant burden on those living with haemophilia and has an impact on broader family, relationships and work. The availability of gene therapy for haemophilia B as a one-time infusion will allow those eligible for treatment to expand their horizons and live life to the full, free from the restrictions of regular injections.’

Hemgenix, also called etranacogene dezaparvovec, was approved for NHS use a year ago but administrative complications slowed its roll out.

The person who received gene therapy was diagnosed with severe haemophilia B aged 18 months. He does not want to be named, but commented: ‘Being free from the burden of knowing I have the condition will be fantastic. To experience life free of the worry and to do things that I wouldn’t normally do will also be amazing. Not needing to plan ahead for treatment deliveries or looking up hospitals in foreign destinations when going on holiday, or having to tell people ‘sorry I can’t do that I’ve got haemophilia’, will be something I’ve always dreamed of.’

Gene therapy was pioneered in the UK by Professor Ted Tuddenham and Professor Amit Nathwani at the Royal Free Hospital in London. The first clinical trials in gene therapy for haemophilia B began in 1999 and this product, which is made by CSL Behring, was approved for use in the NHS in June 2024.

You can find out more about what gene therapy involves here. Talk to you consultant if you would like to know more about this treatment.