Casgevy, a new CRISPR-based gene therapy, has received FDA approval for treating sickle cell disease in patients 12 and older experiencing recurrent crises. This groundbreaking treatment uses a patient's own stem cells, edited to produce fetal hemoglobin, thus preventing the sickling of red blood cells and associated pain. While requiring intensive chemotherapy beforehand, Casgevy offers a potential cure and improved quality of life compared to bone marrow transplants, the previous long-term treatment option. Current research is ongoing to assess the long-term safety and efficacy of Casgevy and to broaden access to this life-changing therapy for all those affected by sickle cell disease. The therapy's success represents significant progress in treating this debilitating inherited blood disorder.

This timeline details the key discoveries leading to the development of CRISPR-Cas9 gene editing. It highlights the contributions of numerous scientists, from the initial observation of CRISPR sequences in bacteria to the experimental demonstration of their function as an adaptive immune system. The timeline traces the crucial steps in understanding the mechanism of CRISPR-Cas9, including the identification of Cas9, guide RNAs, and the PAM sequence. Finally, it culminates in the successful adaptation of CRISPR-Cas9 for genome editing in eukaryotic cells.