‘Much-needed hope' as CRISPR gene editing therapy recommended for sickle cell disease
The UK's National Institute for Health and Care Excellence (NICE) said on Friday that it had approved the use of the gene editing therapy exagamglogene autotemcel (exa-cel) by England's National Health Service (NHS).
NICE had previously rejected the therapy's use on the NHS to treat some people with sickle cell disease in draft guidance released in March. It approved the use of exa-cel for the rare blood disorder beta thalassemia later in the year.
The therapy, which the UK's medicines regulator cleared for use in November 2023, has a list price of £1.6 million (€1.9 million) per course.
Sickle cell disease is a group of inherited blood disorders that affects the shape of red blood cells. People with the condition can experience severe pain, infections, anaemia, and other problems, according to the NHS.
The disease is more common in people from African, Caribbean, Middle Eastern, or South Asian family backgrounds.
There are currently few treatments for symptoms of the disease and the treatments that exist have 'intolerable side effects,' NICE said.
The gene editing therapy will be available to some people aged 12 and older with severe complications from the condition and where a stem cell transplant is 'suitable' but a donor cannot be found, according to the watchdog.
'Much-needed hope'
"The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK,' Funmi Dasaolu, who has sickle cell disease and spoke to the NICE committee, said in a statement.
'It is the beginning of re-addressing the inequalities in care experienced by so many with the condition,' she said, adding that it provides 'much-needed hope'.
The discovery of the CRISPR/Cas9 gene editing tool won two researchers, Emmanuelle Charpentier and Jennifer A. Doudna, the Nobel Prize in Chemistry in 2020.
Exa-cel, also called Casgevy, involves taking a person's blood stem cells, using CRISPR to edit them in a lab, and replacing them in the patient.
'The approval of Exa-cel for NHS use in England is a very exciting moment, not only because this marks the first approval of a CRISPR-based gene therapy for SCD [sickle cell disease] in the NHS, but also because it offers a potentially curative treatment for eligible patients,' said Felicity Gavins, a professor of pharmacology at Brunel University of London.
'However, while Exa-cel is a breakthrough, it is not a cure for all SCD patients, and uncertainties remain about its long-term effectiveness, safety, and accessibility,' Gavins said in a statement.
'It is critical to continue funding research to develop treatment that benefit the broader SCD population and address remaining challenges in care'.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Euronews
2 days ago
- Euronews
Endangered US contraceptives put 1.4 million women at risk, NGO claims
More than 1.4 million women and girls in Africa risk losing access to life-saving care if a stockpile of US-owned contraceptives currently stored in Europe are destroyed in accordance with US government plans, according to NGO International Planned Parenthood Federation (IPPF). The contraceptives, valued at $10 million (€8.5 million ) are currently stored in a warehouse in Belgium, but face destruction following the closure of key American aid agency United States Agency for International Development (USAID), as reported. Plans for them to be incinerated in France in accordance with US government plans have sparked widespread condemnation from civil society organisations and politicians, who are calling for the supplies to be preserved. The IPPF has attempted to purchase the stock from the US government but had its offer rejected. The products were originally intended for distribution to lower-income countries by the now dismantled USAID. Approximately 77% of the stock, many with expiration dates between 2027 and 2029, was intended for use in African nations including the Democratic Republic of the Congo (DRC), Mali, Kenya, Tanzania, and Zambia. 'We are facing a major challenge. The impact of the USAID funding cuts has already significantly affected the provision of sexual and reproductive health services in Tanzania - leading to a shortage of contraceptive commodities, especially implants," said Dr Bakari, Project Coordinator at UMATI, IPPF's Member Association in Tanzania. Tanzania was set to receive more than a million injectable contraceptives and 365,000 implants from the Brussels stockpile, accounting for more than 40% of the total shipment. These supplies represent over half of USAID's annual support to Tanzania's health system and 28% of the country's total annual need. 'In Kenya, the effects of US funding disruptions are already being felt. The funding freeze has caused stock-outs of contraceptives, leaving facilities with less than five months' supply instead of the required 15 months,' said Nelly Munyasia, executive director for the Reproductive Health Network in Kenya also member of IPPF.** She added that the withdrawal of USAID has created a 46% funding gap in Kenya's national family planning programme. The US has long been the largest bilateral donor to family planning, contributing $600 million annually which was 40% of global donor funding. The cancellation of pending procurement contracts has widened an existing global funding gap from $167 million to $210 million across 32 countries, according to the Reproductive Health Supplies Coalition (RHSC), a global partnership of public, private, and non-governmental organisations. The coalition also warned of broader effects of burning this stash. The group said that, when a woman's first choice of contraceptive is missing, she may opt for a less-preferred product, which could lead to a stock-out of that product if demand rises unexpectedly. 'When family planning stocks are compromised, the entire supply chain is at risk, requiring new funding, time, and coordination that cannot materialise at short notice,' they stated. The RHSC has estimated that failure to deliver this stockpile to its intended recipients could result in 362,000 unintended pregnancies, 161,000 unplanned births, and 110,000 unsafe abortions. Political opposition in Europe During a briefing in late July, US State Department spokesperson Thomas Pigott said officials were 'still in the process here in terms of determining the way forward' with regard to the Belgian stock. As the supplies are reportedly set to be transferred to France for destruction, French Green Party politicians have appealed to President Emmanuel Macron to intervene. 'We cannot allow Donald Trump's anti-choice agenda to unfold on our territory. And so today, France must mediate with the Commission,' MEP Mélissa Camara (France/The Greens), one of the signatories of the letter, told Euronews. "Unfortunately there is no legal basis for intervention by a European health authority, let alone the French national drug safety authority, to recover these medical products," the French health ministry told AFP. "Since contraceptives are not drugs of major therapeutic interest, and in this case, we are not facing a supply shortage, we have no means to requisition the stocks," it added. The ministry also said it had no information on where the contraceptives would be destroyed.
LeMonde
01-08-2025
- LeMonde
Contraceptives destroyed in silence: 'Can France accept to become the executor of a senseless policy imposed by the US?'
A stockpile of several million doses of contraceptives, valued at $9.7 million, financed by USAID and usable until 2031, is currently being incinerated in France. These products, originally intended for women in extreme poverty in countries in the Global South, are being destroyed for purely ideological reasons, at the request of the Trump administration. The service provider tasked with the incineration has not yet been identified. This human and environmental waste must be stopped immediately. Why is this happening? Because the US administration, following a blinkered ideological agenda, ordered their destruction as part of the dismantling of USAID. It even refused to transfer the stock to NGOs who were ready to transport and distribute it. This is not a public health issue or a logistical problem. It is a radical political choice. USAID's logistics program, which is usually operated by the company Chemonics, appears to be linked to this operation. However, this cannot be confirmed, since neither the name of the logistics provider nor the name of the incinerator has been made public. The stock, which was previously stored in Belgium, was transported to France in about 10 trucks at the end of July. The exact location of the incineration is deliberately undisclosed. Despite the scale of the operation, the company responsible for handling the medical waste has not been identified. We contacted four major French waste management companies to ask whether they were involved in this operation. All replied that they were not concerned. An economic and environmental absurdity As NGOs active in several African countries, we know exactly what a contraceptive shortage means: unintended pregnancies, abandoned children, unsafe abortions, and girls forced out of school at age 12. It also means women trapped in unwanted motherhood. Can France accept to become the executor of a senseless policy imposed by the US without reaction? This destruction is also an economic and environmental absurdity. It costs more ($167,000) than it would to redistribute contraceptives to countries experiencing shortages. Eliminating usable products that are urgently needed in empty health centers will also generate metric tons of CO₂. It's a total waste.
France 24
25-07-2025
- France 24
World's smallest snake makes big comeback
The Barbados threadsnake (Tetracheilostoma carlae) was found hiding under a rock in central Barbados during an ecological survey in March by the Barbados Ministry of the Environment and National Beautification and conservation group Re:wild. "Barbados threadsnakes are blind snakes, so they're very cryptic," said Connor Blades, a project officer with the Ministry of Environment in Barbados who helped make the finding, in a statement. "They're quite rare also, it seems. There have only been a handful of confirmed sightings since 1889, so there are not many people who have ever seen it, unfortunately." Measuring just three to four inches long (eight to 10 centimeters) when fully grown -- tiny enough to almost fit on a US quarter coin -- the Barbados threadsnake is the world's smallest species of snake. It is distinguished by orange stripes along its back, eyes on the sides of its head and a small scale on its snout. "When you are so accustomed to looking for things and you don't see them, you are shocked when you actually find it," said Justin Springer of Re:wild, who made the discovery alongside Blades. "You can't believe it. That's how I felt. You don't want to get your hopes up too high." The breakthrough came after more than a year of searching, as the pair upturned rocks trapped beneath a tree root. The tiny snake, which was found alongside an earthworm, was taken to the University of West Indies for careful examination under a microscope -- it closely resembles the Brahminy blind snake, an invasive species, so the finding had to be validated -- before it was returned to the forest. Only two percent of the Caribbean island's primary forest remains intact, with the rest cleared for agriculture since the start of the colonial era 400 years ago. The Barbados threadsnake remains particularly vulnerable since it reproduces sexually and females lay a clutch of only one egg. Female Brahminy blind snakes, by contrast, can produce fertile eggs without mating. "The threadsnake's rediscovery is also a call to all of us as Barbadians that forests in Barbados are very special and need protection," said Springer. "Not just for the threadsnake, but for other species as well. For plants, animals and our heritage." © 2025 AFP
