UK Clears Gene-Editing Therapy for Rare Blood Disorder

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Facts

  • The UK's National Health Service (NHS) has been cleared to use gene-editing therapy to treat the blood disorder beta thalassaemia.[1]
  • The therapy, termed Casgevy, may help the estimated 2.3k people in the UK with thalassaemia, a rare group of inherited disorders in which the body produces too little hemoglobin, stunting its ability to carry oxygen.[2][1]
  • The National Institute for Health and Care Excellence has said thalassaemia mainly affects those of Pakistani, Indian, and Bangladeshi origin in the UK. The goal is to replace patients' current need for life-long blood transfusions every three to five weeks.[3]
  • Two firms, Vertex Pharmaceuticals and CRISPR Therapeutics, will offer the therapy involving stem cell transplantation, a Vertex statement said Wednesday.[4]
  • Casgevy reportedly costs more than £1.6M ($2M) per patient, but Vertex has agreed to provide a currently undisclosed discount for up to 460 patients aged12 and above.[5]
  • This is reportedly the first treatment to be licensed on the NHS that uses the gene-editing tool CRISPR. Researchers who invented CRISPR won the 2020 Nobel Prize for chemistry.[6]

Sources: [1]BBC News, [2]Guardian, [3]Nice, [4]The Straits Times, [5]Sky News and [6]AOL.

Narratives

  • Narrative A, as provided by The Indian Express. Gene-editing therapy will revolutionize thalassemia treatment with the UK’s approval of the groundbreaking Casgevy therapy. It corrects the faulty gene responsible for thalassemia, offering a potential lifelong cure. Previously, the only permanent solution was a bone marrow transplant, requiring a closely matched donor. Casgevy, instead, uses the patient’s own stem cells to produce healthy hemoglobin, drastically reducing the need for regular blood transfusions.
  • Narrative B, as provided by Scientific American. As CRISPR receives an outpouring of positive coverage due to its expected ability to cure diseases, it's important not to forget the negative side of the gene-editing industry. Gene-editing, if left unchecked, will undoubtedly go beyond fixing blood diseases and lead to a world where all differences among humans are artificially eradicated. We shouldn't allow the medical industry to play God when it comes to how we're born and how we develop.

Predictions