CRISPR Technology And The Future of Healthcare

Eric Huang, Pharm.D. Candidate 2025

Eric Huang, PharmD Candidate 2025

Have you ever thought about what makes each person so different? Well, it comes down to our genome. Multiple genes code our unique and amazing differences. Unfortunately, those same genes can also be the reason some people are more at risk of inheriting or developing serious genetic conditions. These conditions are life-altering for the patient and their families and it was thought that there would only be a few effective treatments. But what if I told you that two new medications recently got approved to help for the treatment of sickle cell disease, one of the most prominent DNA diseases that has affected up to 100,000 Americans? Well, the dream became a reality on December 8, 2023, when the FDA approved the first cell-based gene therapies to treat sickle cell disease in patients 12 years and older: Casgevy and Lyfgenia. Casgevy uses CRISPR/Cas9, which can directly cut DNA at specific locations. With this, blood stem cells can be modified to increase fetal hemoglobin, a type of hemoglobin that prevents the sickling of the red blood cells. Lyfgenia can modify a patient’s blood stem cells to create HbAT87Q, which lowers the risk of sickling and occlusions. These treatments have completely flipped the narrative on sickle cell disease. A condition that, previously, could only be managed, now has two therapies that can potentially extend the lives  of these patients. It’s an exciting time to see these innovative treatments come to the market and it shows that the industry is well on its way to tackling some of the most difficult and rare conditions that we know of.