First approved CRISPR therapy (Casgevy, sickle-cell disease)
Primera terapia CRISPR aprobada (Casgevy, anemia falciforme)
- Date
- 2023 · confidence high
- Place
- Vertex Pharmaceuticals, Boston, United States, and CRISPR Therapeutics, Zug, Switzerland; approved by the British MHRA in November 2023
- Credited to
- Vertex and CRISPR Therapeutics; first patient Victoria Gray (2019)
- Field
- Life & health
What it is
Editing of a patient's own blood stem cells to reactivate fetal haemoglobin and cure sickle-cell disease and beta-thalassaemia. It was the first CRISPR-based medicine approved anywhere in the world.
What it led to
- Gene editing as real medicine eleven years after its invention
- Two-million-dollar treatments and pay-for-outcome models
- In vivo CRISPR therapies for the liver and heart
Built on
Made possible
- in vivo CRISPR therapies
- editing of haematopoietic stem cells