It’s a historic day for science. The first CRISPR based gene therapy for humans was approved by the FDA this morning. The treatment, named Casgevy, cures sickle-cell disease. Sickle-cell disease is caused by an inherited genetic mutation that made faulty adult hemoglobin, which made red blood cells unhealthy. The therapy itself is very grueling but it offers a complete cure of the disease. It requires weeks of hospitalization to extract sufficient numbers of the patient’s stem cells. The stem cells will then be edited with CRISPR and injected back to the patient. In this particular case, the repressor gene for fetal hemoglobin is cut out with the CRISPR technology, which enables patients to produce healthy fetal hemoglobin to make red blood cells healthy. This WSJ article has a pretty good infographic to explain how Casgevy works. It is amazing!
I wrote about the imminent approval of the first CRISPR based gene therapy three months ago. I now proudly take a victory lap!! I bought CRSP 0.00%↑ shares at ~$50. It went down to below $40 right after I bought it, bounced to $75 before the FDA approval, but tanked to ~$65 after the approval was announced. Anyway, I made some handsome profits here. There is definitely money to be made in biotech stocks but the volatility is not for the faint of heart. Regardless, CRISPR based gene therapy will only progress faster from here and I believe CRISPR technology will be as impactful as deep learning in AI in coming years.