Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA approval for the first CRSPR-based gene therapy treatment for sickle cell anemia (SCA) called Casgevy, developed by Vertex Pharmaceuticals Inc. (NASDAQ: VRTX). Sickle cell is a disease that causes red blood cells to form into a sickle-like shape, which can block blood flow, leading to more serious complications like pain and organ damage. It is caused by a mutation in the hemoglobin gene (HBB). CRISPR-Cas9 acts as molecular scissors that can precisely snip out parts of the HBB genome in living cells. To remedy sickle cells, CRISPR-Cas9 is used to edit genes that inhibit the production of BCL11A, which reactivates rich fetal hemoglobin production to reduce the sickling of blood cells.
