Researchers reviewed what clinical trials involving CRISPR-Cas9 gene editing were registered on the U.S. government's clinicaltrials.gov website as of the end of 2024. They did not look at the results of these trials, but at what diseases the trials were designed to study.
The main finding is that the first therapy using CRISPR technology was approved by the FDA in late 2023. It is approved to treat two serious blood disorders: sickle-cell anemia and transfusion-dependent β-thalassemia. Beyond these approved uses, the registered trials show scientists are exploring whether CRISPR could be used to treat other conditions, including various cancers and infectious diseases.
This review only tells us what research is planned or in progress. It does not provide any results, safety data, or information on how well these experimental treatments might work. The fact that a trial is registered does not mean the treatment is safe or effective. For the one approved therapy, patients should discuss the specific benefits and risks with their doctor, as it is a complex and very new type of treatment.