CRISPR CTX001 Clinical Trial: Promising Results in Sickle Cell Patients
Sickle cell anemia and beta-thalassemia are serious genetic blood disorders, caused by mutations in the gene that produces beta-globin, a component of hemoglobin. People afflicted by either disease suffer from anemia, pain attacks, fatigue, and risk of organ damage, all of which impact their quality of life. Frequent blood transfusions to alleviate symptoms or bone marrow transplants as a long term solution have been the only treatment options so far.
The recent genome engineering revolution has sparked interest among researchers to treat these diseases using gene therapy. As hematopoetic stem cells are relatively accessible forex vivoediting, sickle cell anemia and beta-thalassemia have become prime candidates for gene therapy studies. In our other comprehensive guides, we have covered how CRISPR is being used in sickle cell anemia and beta thalassemia research and treatment.
In this blog post, we specifically report the triumph of CTX001 therapy, a CRISPR-based investigational drug that is being tested in clinical trials for treating sickle cell disease and beta thalassemia. Recent reports indicate that CTX001, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has been effective in restoring hemoglobin levels and alleviating symptoms in patients for more than a year now. Partial data were presented at the American Society of Hematology 2020 conference last week and details of data from two patients were published inthe New England Journal of Medicine.
“It’s something I prayed for my whole life,” she said. “I pray everyone has the same results I did.”- Victoria Gray, one of the trial candidates being treated for sickle cell anemia, told NBC News
