The field of gene therapy has seen unprecedented growth in recent years, with 20 approved indications in the U.S., a record number of approvals in 2023, and a rapid expansion of investigational trials across various diseases. Great strides have been made in potential treatments for inherited and acquired genetic diseases, including hemophilia B, sickle cell…
Adakveo: An FDA-indicated treatment for sickle cell disease
Adakveo (crizanlizumab-tmca) from Novartis is FDA indicated for reducing the number of vaso-occlusive crises in people aged 16 years and older with sickle cell disease. Novartis describes it as “the first and only once-monthly medication to reduce the number of pain crises in sickle cell disease.” The drug works by inhibiting selectin, a group of carbohydrate-binding transmembrane molecules…