Patients with a rare blood disorder in England will become among the first in the world to receive CRISPR-based gene-editing therapy after drugmakers announced a partnership with the National Health Service (NHS).

About 460 patients in England with transfusion-dependent beta thalassaemia – a life-limiting genetic blood disorder – who are 12 or older are potentially eligible for the “life-changing” treatment, the NHS said. The therapy will be offered within weeks.

“This is the latest in a series of revolutionary gene therapies,” NHS Chief Executive Amanda Pritchard said, adding that “this one-off therapy will be fast-tracked to patients who could benefit from the new lease of life it promises".

The new therapy, called Casgevy, is administered as a one-time infusion. Stem cells will be extracted from a patient's bone marrow and brought to a lab, where a gene in the cells will be altered to remove the condition and then infused back into the patient.

It is the first approved treatment using CRISPR technology, which won the Nobel Prize for chemistry in 2020.

'Hope of living longer'

The treatment is intensive. Patients must get chemotherapy first, and then stay in the hospital for four to six weeks to “allow the treated stem cells to embed themselves in the bone marrow and begin producing healthy red blood cells,” the NHS said.

Even so, patients are likely to welcome the change from existing treatments. Most people who could be eligible for Casgevy need blood transfusions every three to five weeks to survive, but in clinical trials for Casgevy, 93 per cent of patients did not need a transfusion for at least a year afterwards, raising researchers’ hopes that it could cure the condition.

“Ordinarily, patients experience painful side effects and undergo regular transfusions which severely impact their quality of life, but this therapy offers people a life free from that as well as the hope of living longer, which is truly amazing news,” Pritchard said.

Beta thalassaemia is a lifelong condition that affects the red blood cells and can lead to anaemia, chronic pain, and mental health problems such as anxiety and depression, according to the NHS. Many patients die in their 50s.

About 2,300 people in the UK have beta thalassaemia, 800 of whom rely on regular blood transfusions, the NHS said.

“I’m only 21 and the idea of depending on transfusions for quite literally the rest of my life is daunting,” Kirthana Balachandran, a medical student from West London who was diagnosed with thalassaemia when she was three months old, said in a statement.

“Not needing to go for regular blood transfusions and worry about side effects or my future health would really change my life.”

Vertex Pharmaceuticals, which developed Casgevy, struck a deal with NHS England to offer the therapy after the National Institute for Health and Care Excellence (NICE) issued positive guidance to recommend its use, the drugmaker said.

"This technology has the potential to be used for treating many other genetic diseases, so this is a really important approval by NICE," Dr James Davies, an associate professor of genomics at the University of Oxford, said in a statement.

Britain's medical regulator also previously authorised Casgevy’s gene-editing therapy to treat sickle-cell disease, which affects about 15,000 people in England.

Vertex said it is working with English authorities to roll out the treatment for sickle-cell patients "as soon as possible" and that it hopes to "rapidly" follow suit in the European Union.

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