The FDA approved the first CRISPR-based gene therapy for sickle cell disease, marking a historic milestone in genetic medicine. The treatment has achieved functional cures in clinical trials.
Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses gene editing to enable patients to produce fetal hemoglobin, compensating for defective adult hemoglobin. Trial participants have remained symptom-free for over two years.
"This is the beginning of a new era in medicine," said CRISPR co-inventor Jennifer Doudna. "We can now directly correct genetic mutations that cause disease rather than just treating symptoms."
The treatment costs $2.2 million per patient, raising accessibility concerns. Insurers are developing payment models to spread costs over time, but many patients in developing countries where sickle cell is most common cannot access the therapy.
Other CRISPR therapies are in development for conditions including beta-thalassemia, certain cancers, and inherited blindness. The technology is progressing faster than regulatory frameworks can adapt.
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