London Daily

Focus on the big picture.
Sunday, Apr 12, 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
Meghan Markle Plans Exclusive Women-Focused Retreat During Australia Visit
Starmer and Trump Hold Strategic Talks on Securing Strait of Hormuz Amid Rising Tensions
Unofficial Australia Visit by Prince Harry and Meghan Expected to Stir Tensions with Royal Circles
Pipeline Attack Cuts Significant Share of Saudi Arabia’s Oil Export Capacity
UK Stocks Rise on Ceasefire Momentum and Renewed Focus on Diplomacy
UK to Hold Further Strategic Talks on Strait of Hormuz Security
Starmer Voices Frustration as Global Tensions Drive Up UK Energy Costs
UK Students Voice Concern Over Proposal for Automatic Military Draft Registration
Rising Volatility Drives Uncertainty in UK Fuel and Petrol Prices
UK Moves to Deploy ‘Skyhammer’ Anti-Drone System to Strengthen Airspace Defense
New Analysis Explores UK Budget Mechanics in ‘Behind the Blue’ Feature
Man Arrested After Four Die in Channel Crossing Tragedy
UK Tightens Immigration Framework with New Sponsor Rules and Fee Increases
UK Foreign Secretary Highlights Impact of Intensified Strikes in Lebanon
UK Urges Inclusion of Lebanon in US-Iran Ceasefire Framework
UK Stocks Ease as Ceasefire Doubts in Middle East Weigh on Investor Confidence
UK Reassesses Cloud Strategy Amid Criticism Over Limited Support Measures
UK Calls for Full and Toll-Free Access Through Strait of Hormuz Amid Rising Tensions
Starmer Signals Strategic Shift for Britain Amid Escalating Iran-Linked Tensions
UK Issues Firm Warning to Russia Over Covert Underwater Military Activity
OpenAI Halts Stargate UK Project, Casting Uncertainty Over Britain’s AI Expansion Plans
Starmer Voices Frustration Over Global Pressures Driving UK Energy Costs Higher
UK Deploys Military Assets to Protect Undersea Cables From Suspected Russian Threat
Canada Aligns With US, UK and Australia as Europe Prepares Major Digital Border Overhaul
Meghan Markle’s Planned Australia Appearance Sparks Fresh Speculation
Starmer Warns Sustained Effort Needed to Ensure US–Iran Ceasefire Holds
UK to Partner with Shipping Industry to Rebuild Confidence in Strait of Hormuz, Cooper Says
UK Interest Rate Expectations Ease Following US–Iran Ceasefire Agreement
Starmer Signals Major Effort Needed to Fully Reopen Strait of Hormuz During Gulf Visit
UK Fuel Prices Face Ongoing Volatility Amid Global Pressures and Domestic Factors
Kanye West’s Planned Italy Festival Appearance Draws Debate After UK Entry Ban
Smuggling Routes Shift Toward Belgium as Migrant Crossings to UK Evolve
Ceasefire Offers Potential Relief for UK Fuel and Food Prices Amid Ongoing Uncertainty
Iran Conflict Raises Questions Over UK’s Global Influence and Military Preparedness
Senator McConnell Visits Kentucky to Highlight Federal Investment in Local Projects
Kanye West Barred from Entering UK as Legal Grounds Come into Focus
UK Denies Visa to Kanye West After Sponsors Withdraw from Wireless Festival
Trump-Era Forest Service Restructuring Leads to Closure of UK Lab Focused on Kentucky Woodland Health
Foreign Students in the UK Describe Harsh Living Conditions and Financial Pressures
Reform UK Proposes Visa Restrictions on Nations Pursuing Reparations Claims
Public Reaction Divides Over UK Decision to Bar Kanye West
Calls Grow for UK to Review US Base Access Following Concerns Over Escalating Rhetoric
UK Indicates It Will Not Permit Use of Its Bases for Potential US Strikes on Iran’s Energy Infrastructure
UK Prime Minister Defends Decision to Bar Kanye West, Questions Festival Booking
UK Accelerates Efforts to Harmonise Medical Technology Rules with United States
Wireless Festival Cancelled After Kanye West Denied Entry to the United Kingdom
Australia’s most decorated living soldier was arrested at Sydney Airport and charged with five counts of war-crime murder for the killing of unarmed Afghan civilians
The CIA’s Secret Technology That Can Find You by Your Heartbeat Successfully Locates Downed Airman
Operation Europe: Trump Deploys Vance to Hungary to Save the EU
King Charles Faces Criticism From Some UK Christians Over Absence of Easter Message
×