Skip to content
Search

Latest Stories

MHRA approves first ‘gene-editing’ treatment for sickle-cell disease and thalassemia

MHRA approves first ‘gene-editing’ treatment for sickle-cell disease and thalassemia

It can restore healthy haemoglobin production in patients and free them from painful symptoms  

The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia.


Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over.

To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal.

The MHRA’s authorisation came after a rigorous assessment of its safety, quality and effectiveness.

Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA, informed that Casgevy has been found to “restore healthy haemoglobin production” in the majority of study participants, relieving their symptoms.

“The MHRA will continue to closely monitor the safety and effectiveness of Casgevy, through real-world safety data and post-authorisation safety studies being carried out by the manufacturer,” he said.

How the gene therapy work?

Both sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, a protein used by red blood cells to carry oxygen around the body.

This genetic error can lead to attacks of very severe pain, serious and life-threatening infections, and anaemia in people with sickle cell disease.

People with β-thalassaemia can experience severe anaemia, and they may need a blood transfusion every 3 to 5 weeks, and injections and medicines throughout their lives.

Casgevy is designed to treat these conditions by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

First, stem cells are taken out of the patient’s bone marrow, edited in a laboratory and then infused back into the patient.

As stated by the MHRA, the results of this treatment have the potential to be life-long, which is good news for patients suffering from these genetic conditions.

More For You

The Complete Anti-Inflammatory Guide - a useful resource for pharmacists to support patient health and wellbeing

Chronic inflammation is linked to more than 50 per cent of all deaths worldwide and one in five cancers.

The Complete Anti-Inflammatory Guide - a useful resource for pharmacists to support patient health and wellbeing

When Hemant Patel was struck down by Covid, he was determined to discover what was it that led to the disease having a devastating impact on the lives of people who seemed be healthy – the culmination of his journey is the book, The Complete Anti-Inflammatory Guide.

“I was gasping for breath and counting the number of days I might be alive when I was infected with Covid,” reveals Patel.

Keep ReadingShow less
Cargo plane unloading pharmaceuticals at US airport amid tariff concerns and stockpiling

Imports jumped in particular from Ireland, the top drug exporter to the US

Pic credit: iStock

Pharma imports to US surged in March as drugmakers look to avoid tariffs

Pharmaceutical imports to the US surged in March as drugmakers stocked up ahead of potential US tariffs on their products, which have historically been exempt from such fees.

Total imports of pharmaceutical products exceeded $50 billion in the month - the equivalent of 20 per cent of all pharmaceutical imports in 2024, according to data from a U.S. Commerce Department report on Tuesday.

Keep ReadingShow less
Scottish community pharmacist working in high street pharmacy with supportive environment

The PDA wants investment to reach those who deliver services to patients every day

Pic credit: iStock

Funding boost in Scotland “needs to work for not just pharmacy owners but pharmacists too”, says PDA

An increase in funding for community pharmacies in Scotland needs to lead to a greater focus on supporting pharmacists rather than “simply sustain business models”, according to the Pharmacy Defence Association (PDA).

Earlier this week, Community Pharmacy Scotland (CPS) accepted the Scottish government’s initial financial offer for the 2025/26 fiscal year, securing a guaranteed minimum reimbursement of £120 million for community pharmacies — up from £110 million from 2024/25.

Keep ReadingShow less
GP surgery upgrades for annual appointments

The surgeries will have additional space to “see more patients, boost productivity and improve patient care”

Pic credit: iStock

GP surgery upgrades to create 8.3 million more annual appointments

Over 1,000 GP surgeries will have their premises modernised to meet the needs of a further 8.3 million appointments each year, the government has announced.

Backed by a cash injection of over £102 million, the surgeries will have additional space to “see more patients, boost productivity and improve patient care”.

Keep ReadingShow less
Wales boosts funding for pharmacy-led UTI and sore throat test services

The sore throat test (STTT) and treat service will be widely available

Pic credit: istock

Welsh pharmacies receive funding boost for clinical services

Two key clinical services will be available in 99 per cent of community pharmacies across Wales after a boost in funding.

The sore throat test (STTT) and treat service and the urinary tract infection (UTI) service have both benefitted from contractual negotiations between the Welsh Government and Community Pharmacy Wales (CPW).

Keep ReadingShow less