London Daily

Focus on the big picture.
Wednesday, Jan 21, 2026

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
Prince William to Make Official Visit to Saudi Arabia in February
Prince Harry Breaks Down in London Court, Says UK Tabloids Have Made Meghan Markle’s Life ‘Absolute Misery’
Malin + Goetz UK Business Enters Administration, All Stores Close
EU and UK Reject Trump’s Greenland-Linked Tariff Threats and Pledge Unified Response
UK Deepfake Crackdown Puts Intense Pressure on Musk’s Grok AI After Surge in Non-Consensual Explicit Images
Prince Harry Becomes Emotional in London Court, Invokes Memory of Princess Diana in Testimony Against UK Tabloids
UK Inflation Rises Unexpectedly but Interest Rate Cuts Still Seen as Likely
Buying an Ally’s Territory: Strategic Genius or Geopolitical Breakdown?
AI Everywhere: Power, Money, War, and the Race to Control the Future
Trump vs the World Order: Disruption Genius or Global Arsonist?
Trump vs the World Order: Disruption Genius or Global Arsonist?
Trump vs the World Order: Disruption Genius or Global Arsonist?
Trump vs the World Order: Disruption Genius or Global Arsonist?
Arctic Power Grab: Security Chessboard or Climate Crime Scene?
Starmer Steps Back from Trump’s ‘Board of Peace’ Amid Strained US–UK Relations
Prince Harry’s Lawyer Tells UK Court Daily Mail Was Complicit in Unlawful Privacy Invasions
UK Government Approves China’s ‘Mega Embassy’ in London Amid Debate Over Security and Diplomacy
Trump Cites UK’s Chagos Islands Sovereignty Shift as Justification for Pursuing Greenland Acquisition
UK Government Weighs Australia-Style Social Media Ban for Under-Sixteens Amid Rising Concern Over Online Harm
Trump Aides Say U.S. Has Discussed Offering Asylum to British Jews Amid Growing Antisemitism Concerns
UK Seeks Diplomatic De-escalation with Trump Over Greenland Tariff Threat
Prince Harry Returns to London as High Court Trial Begins Over Alleged Illegal Tabloid Snooping
High-Speed Train Collision in Southern Spain Kills at Least Twenty-One and Injures Scores
Meghan Markle May Return to the U.K. This Summer as Security Review Advances
Trump’s Greenland Tariff Threat Sparks EU Response and Risks Deep Transatlantic Rift
Prince Harry’s High Court Battle With Daily Mail Publisher Begins in London
Trump’s Tariff Escalation Presents Complex Challenges for the UK Economy
UK Prime Minister Starmer Rebukes Trump’s Greenland Tariff Strategy as Transatlantic Tensions Rise
Prince Harry’s Last Press Case in UK Court Signals Potential Turning Point in Media and Royal Relations
OpenAI to Begin Advertising in ChatGPT in Strategic Shift to New Revenue Model
GDP Growth Remains the Most Telling Barometer of Britain’s Economic Health
Prince William and Kate Middleton Stay Away as Prince Harry Visits London Amid Lingering Rift
Britain Braces for Colder Weather and Snow Risk as Temperatures Set to Plunge
Mass Protests Erupt as UK Nears Decision on China’s ‘Mega Embassy’ in London
Prince Harry to Return to UK to Testify in High-Profile Media Trial Against Associated Newspapers
Keir Starmer Rejects Trump’s Greenland Tariff Threat as ‘Completely Wrong’
Trump to hit Europe with 10% tariffs until Greenland deal is agreed
Prince Harry Returns to UK High Court as Final Privacy Trial Against Daily Mail Publisher Begins
Britain Confronts a Billion-Pound Wind Energy Paradox Amid Grid Constraints
The graduate 'jobpocalypse': Entry-level jobs are not shrinking. They are disappearing.
Cybercrime, Inc.: When Crime Becomes an Economy. How the World Accidentally Built a Twenty-Trillion-Dollar Criminal Economy
The Return of the Hands: Why the AI Age Is Rewriting the Meaning of “Real Work”
UK PM Kier Scammer Ridicules Tories With "Kamasutra"
Strategic Restraint, Credible Force, and the Discipline of Power
United Kingdom and Norway Endorse NATO’s ‘Arctic Sentry’ Mission Including Greenland
Woman Claiming to Be Freddie Mercury’s Secret Daughter Dies at Forty-Eight After Rare Cancer Battle
UK Launches First-Ever ‘Town of Culture’ Competition to Celebrate Local Stories and Boost Communities
Planned Sale of Shell and Exxon’s UK Gas Assets to Viaro Energy Collapses Amid Regulatory and Market Hurdles
UK Intensifies Arctic Security Engagement as Trump’s Greenland Rhetoric Fuels Allied Concern
Meghan Markle Could Return to the UK for the First Time in Nearly Four Years If Security Is Secured
×