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Historic world first approval: CRISPR to treat genetic diseases

The landmark approval could transform the treatment of sickle-cell disease and β-thalassaemia — but the technology comes at a considerable cost

Thu. 16 November 2023


In a groundbreaking move, the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of CRISPR gene editing technology to treat diseases, setting a significant milestone in biotechnology. The approved therapy—Casgevy, targets blood conditions such as sickle-cell disease and β-thalassaemia, offering a potential paradigm shift in their treatment. However, the efficacy comes at a cost, as the cutting-edge technology proves to be expensive.

Casgevy, developed by Vertex Pharmaceuticals in Boston, Massachusetts, and CRISPR Therapeutics in Zug, Switzerland, has received approval based on promising results from clinical trials. Administered through intravenous infusion, the one-time treatment demonstrated remarkable outcomes in alleviating the symptoms of sickle-cell disease and β-thalassaemia.

For sickle-cell disease, 28 out of 29 participants experienced complete relief from debilitating pain for at least one year post-treatment. In the case of severe β-thalassaemia, where conventional treatment involves monthly blood transfusions, 93% of the 54 participants did not require red-blood-cell transfusions for at least a year after receiving Casgevy.

The gene therapy leverages the Nobel Prize-winning CRISPR technology, targeting errors in the DNA sequence responsible for haemoglobin production. Both sickle-cell disease and β-thalassaemia result from these genetic anomalies, leading to abnormal haemoglobin and severe health issues.

Casgevy uses CRISPR to edit genes encoding for haemoglobin in blood-producing stem cells taken from the bone marrow of patients. The edited cells, once infused back into the body, produce fetal haemoglobin, mitigating the abnormalities associated with adult haemoglobin in affected individuals.

While the treatment shows promise, safety concerns persist. Participants in ongoing trials experienced side effects like nausea, fatigue, fever, and an increased infection risk. The potential for unintended genetic modifications, a known challenge with CRISPR, remains a point of cautious consideration among experts.

As the US Food and Drug Administration contemplates the approval of Casgevy for sickle-cell disease, and the European Medicines Agency reviews it for both diseases, the therapy's accessibility remains a concern. The intricate process of obtaining and editing patients' blood stem cells makes it challenging to implement in low- and middle-income countries, limiting its reach to nations with advanced healthcare systems.

Moreover, the exorbitant cost of Casgevy raises questions about global accessibility. Estimated at around $2 million per patient, the therapy's high price tag emphasizes the need to address affordability and equitable access. As the UK establishes reimbursement and access frameworks, the global community awaits further developments in the pursuit of this groundbreaking CRISPR treatment.

What is CRISPR?

According to the genetics glossary of National Human Genome Research Institute  "CRISPR (short for “clustered regularly interspaced short palindromic repeats”) is a technology that research scientists use to selectively modify the DNA of living organisms."

Modifying the genes inside the cells was always thought to be time-consuming, difficult and sometimes impossible. However, CRISPR/Cas9 genetic scissors are making it possible to change the life code over the course of a few weeks.

Using CRISPR as genetic scissors researchers can change the DNA of living organisms with extremely high precision, because of which it is making a revolutionary impact on the life sciences, by helping develop new cancer therapies and making the dream of curing genetic diseases come true.

Two scientists— Emmanuelle Charpentier and Jennifer A. Doudna— who pioneered the revolutionary CRISPR gene-editing technology won the Nobel Prize in Chemistry in 2020.

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