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Case study: Portsmouth born man with Haemophilia-B finds new lease of life after taking part in a gene therapy clinical research study

John decided to sign up for the HOPE-B study in 2019

An NHS patient with Haemophilia-B has been “functionally cured” of the condition, after taking part in a study of a new treatment. 

John Curley, 52, was one of just over 50 participants who took part in a landmark gene therapy trial, which was supported by the National Institute of Health and Care Research (NIHR). 

He said thanks to the therapy he is now able to live a “near normal life”. 

John was born with severe Haemophilia-B, a rare genetic disorder that prevents the blood from clotting. Even a small cut can be incredibly dangerous to someone living with the condition as a wound will continue to bleed.

Management of the condition in the past was very different to today. John explained that he would regularly visit the hospital to have the clotting protein replaced through a drip. Later in his adolescence, advances in medicine meant that the drug could be self-administered by injections at home.

In 2019, John was invited to take part in the HOPE-B Study under uniQure and CSL Behring, running at University Hospital Southampton NHS Foundation Trust. As part of the study, John received a single infusion of an experimental gene therapy, which aimed to provide patients with a new “working copy” of the gene which is faulty in haemophilia B to enable the body to produce its own clotting factor protein. 

The goal of factor IX gene therapy is to provide people with the ability to make enough functional factor IX to reduce or even eliminate the need for exogenous factor IX injections for many years. This is what doctors would refer to as a “functional cure”.  The study found that following gene therapy 96% of patients were able to stop their regular factor IX prophylactic injections. Experts say advances like this could make a huge difference for haemophilia patients in the future.

In John’s childhood, he was restricted in playing any physical sports such as rugby or football. He said: “The condition always made me aware of my surroundings and I think that could make me quite timid sometimes, I would avoid any confrontation. 

“On the social side it could be quite difficult as well. When I was a teenager some of my mates went travelling around the world and I can never do that. I always felt that I had been left out of things, it did affect me.”

Today, John enjoys playing football, riding his motorbike and is able to go on holiday without having to worry about insurance or travelling with large volumes of medication.

The groundbreaking results offer the potential for a “functional cure” for eligible people with haemophilia B, which was John’s motivation for taking part in research. 

He explains: “I have three sons but often think about what would’ve happened if I had a daughter. She would have inherited the abnormal factor IX gene and therefore been a carrier for the disease. If she’d have gone on to have a son, there would have been a chance that he would have haemophilia”. 

“I think I would have had major guilt for passing the gene on. It's amazing to think that worry could be eradicated soon and that something I was told was incurable can now, be effectively managed with a single-dose treatment.”

Following the hour-long infusion, taking part in the study involved regular reporting of symptoms. John described it as “No big deal.”

He said: “I trusted the consultants and it felt nice to be able to give something back, you know?”

You can watch a YouTube video of John's story on the Clinical Research | Southampton YouTube channel. This YouTube channel is run by University Hospital Southampton NHS Foundation Trust, in partnership with the University of Southampton.

Dr Rashid Kazmi, Principal Investigator of the HOPE-B study at UHS, said, “The gene therapy for adults with severe or moderately severe haemophilia B has been approved in the United States by the Food and Drug Administration (FDA) in November 2022 and European Commission has granted a conditional marketing authorization in February 2023. The Medicines and Healthcare products Regulatory Agency (MHRA) decision is expected shortly”

“It is however still undecided which cohort of haemophilia patients would be selected to receive the drug and which sites across the UK would provide it.”

The trial was led by researchers at University Hospital Southampton NHS Foundation Trust (UHS) and the results will be published in the New England Journal of Medicine.

To find out more about potential research opportunities and studies taking place near you, visit