UK Becomes First Country to Approve Crispr Gene Editing Therapy

The first-of-its-kind gene-editing tool for sickle cell disease was accelerated to market through a regulatory initiative.

A general view of staff on a NHS hospital ward at Ealing Hospital in London, on Jan. 18, 2023. Jeff Moore/PA Media

The UK has become the first country to regulate the authorisation of a CRISPR-based gene-editing therapy in the world.

On Thursday, the government announced that new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over.

Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, a technology that edits genes by “precisely cutting DNA and then harnessing natural DNA repair processes to modify the gene in the desired manner.”

Sickle Cell Disease and β-Thalassemia

The government said that both sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.

It noted that sickle cell disease is particularly common in people with an African or Caribbean family background. β-thalassemia mainly affects people of Mediterranean, south Asian, southeast Asian and Middle Eastern origin.

Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. To do this, stem cells are taken out of bone marrow, edited in a laboratory and then infused back into the patient after which the results have the potential to be life-long.

Casgevy was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.  The ILAP is an initiative to accelerate the timeline for new drugs to get marketing authorisation in the UK.

After Brexit, regulatory oversight switched from the European Medicines Agency (EMA) to the MHRA.

Partners Vertex Pharmaceuticals and CRISPR Therapeutics celebrated the authorisation.

Casgevy, also knows as, Exa-cel is also under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. Food and Drug Administration (FDA).

“Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world,” said Reshma Kewalramani, M.D., chief executive officer and president of Vertex.

‘Enabler’

There are concerns that the regulator is at risk of moving from watchdog to becoming an “enabler” of the pharmaceutical industry because of UK of a pledge to automatically sign off medicines.

In March, Chancellor Jeremy Hunt announced that £10 million will be given to the UK regulator, the MHRA, to help streamline approvals for new medical products.

Mr. Hunt said: “We are lucky with the MHRA to have one of the most respected drugs regulators in the world, indeed the very first to licence a COVID vaccine.

“From 2024, they will move to a different model which will allow rapid, often near automatic sign-off for medicines and technologies already approved by trusted regulators in other parts of the world such as the United States, Europe or Japan.

“At the same time from next year, they will set up a swift new approval process for the most cutting-edge medicines and devices to ensure the UK becomes a global centre for their development,” he added.

At the time, consultant pathologist Dr. Clare Craig told The Epoch Times, ’that’s not what the public needs them for, the public needs them to be a watchdog.”

Dr. Craig said that there isn’t a campaign where patients are saying things are moving too slowly.

“It doesn’t mean that patients can’t get drugs that they need, because doctors are able to prescribe things off-license if the circumstances mean that that’s a sensible thing to do,” she added.

“It’s being spun as if there are all these patients who are desperate to have drugs and they just, they miss out on them because it wasn’t approved in a 100 days and that’s just a fancy really, that’s not the issue,” she said.