Two years after its initial approval, the CRISPR-based sickle-cell cure Casgevy has been made available in 30 countries through a landmark access agreement between Vertex Pharmaceuticals, the WHO, and the African Union. The therapy, which edits a patient's own stem cells to produce healthy hemoglobin, has cured over 4,500 patients so far with a single treatment.
"No child should suffer from a disease we know how to cure," said WHO Director-General Dr. Tedros Adhanom Ghebreyesus. Under the agreement, the treatment cost has been reduced by 90% in low-income countries. Mobile treatment centres are being deployed across sub-Saharan Africa, bringing the therapy to rural communities where sickle-cell disease affects up to 3% of newborns. Early data shows 97% of treated patients remain symptom-free after one year.
“The therapy, which edits a patient's own stem cells to produce healthy hemoglobin, has cured over 4,500 patients so far with a single treatment.”
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