CRISPR/Cas9: edição do DNA e o tratamento de doenças
Em outubro de 2020, o mundo conheceu os vencedores do prêmio Nobel de química de 2020: duas cientistas, Dra. Jennifer Doudna e Dra. Emmanuelle Charpentier ...
Reino Unido
Em novembro e dezembro de 2023, os órgãos competentes de saúde do <anchor>Reino Unido</anchor> e dos Estados Unidos, respectivamente, aprovaram um tratamento para anemia falciforme (e beta-talassemia no caso do Reino Unido) com base em CRISPR-Cas9. No entanto, não são todos os pacientes que podem se submeter a esses tratamentos, pois existem vários requisitos que devem ser seguidos para que esses sejam elegíveis ao tratamento.
The world’s first CRISPR therapy is approved: who will receive it?
The world’s first CRISPR therapy is approved: who will receive it?

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- 21 November 2023

# The world’s first CRISPR therapy is approved: who will receive it?

The go-ahead for Vertex’s gene editing therapy in sickle cell disease and β-thalassemia is a historic milestone, but this one-time treatment is costly.


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Misshapen red blood cells clump and clog blood vessels in sickle cell disease, depriving tissues of oxygen and causing severe pain. Credit: H.S. Photos / Alamy Stock Photo

The UK’s regulator has approved the world’s first CRISPR–Cas9 gene editing therapy, which aims to cure sickle cell disease and transfusion-dependent β-thalassemia. Casgevy (exagamglogene autotemcel) is a first-of-its-kind treatment made by Vertex Pharmaceuticals and CRISPR Therapeutics in Zug, Switzerland. It comes just 11 years after Jennifer Doudna and Emmanuelle Charpentier [invented the technology](https://doi.org/10.1126/science.1225829). The green light from the Medicines and Healthcare Products Agency represents a major scientific achievement for Vertex and CRISPR and a landmark for the biotech industry.

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_Nature Biotechnology_ **42**, 3-4 (2024)

_doi: https://doi.org/10.1038/d41587-023-00016-6_

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