London Daily

Focus on the big picture.
Sunday, Dec 14, 2025

Transformational therapy cures haemophilia B

Transformational therapy cures haemophilia B

A "transformational" therapy has effectively cured people with the bleeding disorder haemophilia B, say British doctors.

The treatment corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding.

Elliott Mason, who was part of the trial which tested the therapy, says his life now feels "completely normal".

The medical team says the majority of adults with haemophilia could be cured in the next three years.

From the day Elliott was born, he was unable to make enough of a crucial protein called clotting factor IX. When you cut yourself and it scabs over, clotting factor IX is one of the proteins that stops the bleeding.

It meant Elliott grew up "anxious of getting hurt", and teachers "wrapped me up in bubble wrap". The sport he wanted to play - rugby - was completely off limits.

"I didn't like the fact that I was different and not able to do things," he says.

At one point, Elliott was having injections of factor IX every other day in order to prevent a deadly bleed.

But he managed to stay healthy, unlike many others with haemophilia who face severe damage to their joints from the bleeding.

"We have a lot of young patients in excruciating agony and there's nothing we can do to reverse the joint damage," says Prof Pratima Chowdary, from the Royal Free Hospital and University College London.


Gene therapy


Elliot was given an engineered virus that was filled with the instructions for manufacturing the missing factor IX.

The virus acts like a microscopic postman that delivers the blueprints to the liver, which then starts producing the clotting protein.

It was a one-off infusion that took about an hour to drip into Elliott's body.

He recalls being "astonished" to see the amount of factor IX in his blood go from only 1% of normal levels to normal.

Elliott says the treatment has made his life "completely normal", without him having to worry how haemophilia could affect it


The findings, published in the New England Journal of Medicine, showed nine out of 10 patients given the therapy no longer needed their clotting factor IX injections.

"I've not had any treatment since I had my therapy, it's all a miracle really, well it's science, but it feels quite miraculous to me," says Elliott, who now lives in London.

"My life is completely normal, there's nothing that I have to stop and think 'how might my haemophilia affect this?'."

For Elliott, that includes skiing and riding a motorbike.


'Very excited'


"We're very excited by the results," said Prof Chowdary.

She says there was a "transformational impact" about a year after the therapy, when suddenly people realised: "I don't need to worry about my haemophilia at all."

This trial is just the latest in a series of breakthroughs in treating both haemophilia A and B.

Prof Chowdary told me she's now "looking for my next job", as curing haemophilia "will be a reality for the majority of the adults in the next one to three years."

But there are still questions which need answering:

*  How much will the gene therapies cost? Current clotting factor injections can cost between £150,000 and £200,000 per patient per year

*  How long will the treatment last? Studies suggest it will be at least a decade, but nobody knows for sure

*  How early in life can the therapy be given? Before the age of 12 the liver is still developing, but scientists hope it will be an option from then on

Clive Smith, chairman of the Haemophilia Society, said: "This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments.

"If they are shown to be safe and effective, NICE [National Institute for Health and Care Excellence] and the NHS must work together to make these innovative treatments available."

Newsletter

Related Articles

0:00
0:00
Close
UK Government Declines to Comment After ICC Prosecutor Alleges Britain Threatened to Defund Court Over Israel Arrest Warrant
Apple Shutters All Retail Stores in the United Kingdom Under New National COVID-19 Lockdown
US–UK Technology Partnership Strains as Key Trade Disagreements Emerge
UK Police Confirm No Further Action Over Allegation That Andrew Asked Bodyguard to Investigate Virginia Giuffre
Giuffre Family Expresses Deep Disappointment as UK Police Decline New Inquiry Into Andrew Mountbatten-Windsor Claims
Transatlantic Trade Ambitions Hit a Snag as UK–US Deal Faces Emerging Challenges
Ex-ICC Prosecutor Alleges UK Threatened to Withdraw Funding Over Netanyahu Arrest Warrant Bid
UK Disciplinary Tribunal Clears Carter-Ruck Lawyer of Misconduct in OneCoin Case
‘Pink Ladies’ Emerge as Prominent Face of UK Anti-Immigration Protests
Nigel Farage Says Reform UK Has Become Britain’s Largest Party as Labour Membership Falls Sharply
Google DeepMind and UK Government Launch First Automated AI Lab to Accelerate Scientific Discovery
UK Economy Falters Ahead of Budget as Growth Contracts and Confidence Wanes
Australia Approves Increased Foreign Stake in Strategic Defence Shipbuilder
Former UK Prime Minister Boris Johnson proclaims, “For Ukraine, surrendering their land would be a nightmare.”
Microsoft Challenges £2.1 Billion UK Cloud Licensing Lawsuit at Competition Tribunal
Fake Doctor in Uttar Pradesh Accused of Killing Woman After Performing YouTube-Based Surgery
Hackers Are Hiding Malware in Open-Source Tools and IDE Extensions
Traveling to USA? Homeland Security moving toward requiring foreign travelers to share social media history
UK Officials Push Back at Trump Saying European Leaders ‘Talk Too Much’ About Ukraine
UK Warns of Escalating Cyber Assault Linked to Putin’s State-Backed Operations
UK Consumer Spending Falters in November as Households Hold Back Ahead of Budget
UK Orders Fresh Review of Prince Harry’s Security Status After Formal Request
U.S. Authorises Nvidia to Sell H200 AI Chips to China Under Security Controls
Trump in Direct Assault: European Leaders Are Weak, Immigration a Disaster. Russia Is Strong and Big — and Will Win
"App recommendation" or disguised advertisement? ChatGPT Premium users are furious
"The Great Filtering": Australia Blocks Hundreds of Thousands of Minors From Social Networks
Mark Zuckerberg Pulls Back From Metaverse After $70 Billion Loss as Meta Shifts Priorities to AI
Nvidia CEO Says U.S. Data-Center Builds Take Years while China ‘Builds a Hospital in a Weekend’
Indian Airports in Turmoil as IndiGo Cancels Over a Thousand Flights, Stranding Thousands
Hollywood Industry on Edge as Netflix Secures Near-$60 Bln Loan for Warner Bros Takeover
Drugs and Assassinations: The Connection Between the Italian Mafia and Football Ultras
Hollywood megadeal: Netflix acquires Warner Bros. Discovery for 83 billion dollars
The Disregard for a Europe ‘in Danger of Erasure,’ the Shift Toward Russia: Trump’s Strategic Policy Document
Two and a Half Weeks After the Major Outage: A Cloudflare Malfunction Brings Down Multiple Sites
UK data-regulator demands urgent clarity on racial bias in police facial-recognition systems
Labour Uses Biscuits to Explain UK Debt — MPs Lean Into Social Media to Reach New Audiences
German President Lays Wreath at Coventry as UK-Germany Reaffirm Unity Against Russia’s Threat
UK Inquiry Finds Putin ‘Morally Responsible’ for 2018 Novichok Death — London Imposes Broad Sanctions on GRU
India backs down on plan to mandate government “Sanchar Saathi” app on all smartphones
King Charles Welcomes German President Steinmeier to UK in First State Visit by Berlin in 27 Years
UK Plans Major Cutback to Jury Trials as Crown Court Backlog Nears 80,000
UK Government to Significantly Limit Jury Trials in England and Wales
U.S. and U.K. Seal Drug-Pricing Deal: Britain Agrees to Pay More, U.S. Lifts Tariffs
UK Postpones Decision Yet Again on China’s Proposed Mega-Embassy in London
Head of UK Budget Watchdog Resigns After Premature Leak of Reeves’ Budget Report
Car-sharing giant Zipcar to exit UK market by end of 2025
Reports of Widespread Drone Deployment Raise Privacy and Security Questions in the UK
UK Signals Security Concerns Over China While Pursuing Stronger Trade Links
Google warns of AI “irrationality” just as Gemini 3 launch rattles markets
Top Consultancies Freeze Starting Salaries as AI Threatens ‘Pyramid’ Model
×