Five New Gene Therapies For Sickle Cell Disease Currently In Clinical Trials

Despite being one of the world’s most common inherited blood disorders, sickle cell disease has few treatment options, even with the approval of three new drugs since 2017. Several companies hope to use new gene-editing tools to cure the disease by targeting the mutations that cause it. One such treatment is in the final stage of human testing and may be submitted for approval in late 2022 or early 2023. While candidates in the pipeline show great promise for a gene-based treatment for sickle cell disease, the challenges facing the practical use of any such innovations may get in the way of changing the global impact of this disease.