The UK has become the first country in the world to approve CRISPR gene-editing therapy.
Regulators approved the use of CRISPR for the treatment of inherited diseases sickle-cell anaemia and -thalassaemia on Thursday.
The former affects the shape of red blood cells of 20 million people worldwide and can cause debilitating pain.
About 80 to 90 million people carry some version of -thalassaemia worldwide.
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.
These are repetitive DNA sequences in the genomes of organisms such as bacteria.
The bacteria transcribe these double stranded DNA elements to single-stranded RNA upon a viral infection.
The cell can then rewrite the code as it fixes the cut.
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The specific product from the trials is Casgevy.
The companies behind Casgevy have yet to disclose the price of the therapy.
The European Medicines Agency (EMA) is reportedly also reviewing the treatment for both diseases.
Story byLinnea Ahlgren
Linnea is the senior editor at TNW, having joined in April 2023.
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