Marisa Wexler, MS, senior science writer —

Treatment with the gene-editing therapy Casgevy (exagamglogene autotemcel) substantially decreased the rates of painful vaso-occlusive crises (VOCs) among people with sickle cell disease taking part in a Phase 2/3 clinical trial. Full results from the trial, which supported the recent approvals of Casgevy in the U.S.,…

England’s National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending the country’s National Health Service (NHS) cover Pfizer’s Oxbryta (voxelotor) to treat sickle cell disease. “Today’s approval by NICE marks a significant milestone in sickle cell care, addressing decades of limited access to…

A nurse clinician-scientist and professor at the University of Toronto, in Canada, is being honored for her work in developing iCanCope, a mobile app that aims to help in sickle cell disease (SCD) pain management. Jennifer Stinson, PhD, was awarded the 2023 Barer-Flood Prize from the Canadian Institutes…

The American Red Cross and the Sickle Cell Disease Association of Illinois (SCDAI) recently joined a national distributor of propane, natural gas, and other energy-related products to host a blood donation event to raise awareness about sickle cell disease and encourage blood donations to support transfusions for sickle cell…

Bluebird Bio has struck a deal with Medicaid in the state of Michigan to allow eligible people with sickle cell disease access to the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel). The new deal will provide Lyfgenia through an outcomes-based agreement — a financial structure in…

Starting in 2025, the U.S. Department of Health and Human Services (HHS) will introduce a pricing model that aims to make new cell and gene therapies more accessible for people with sickle cell disease who are on Medicaid, the health program for lower income residents jointly funded by the…

England’s National Health Service (NHS) is introducing a new genetic test that seeks to make it safer for people with sickle cell disease and other blood disorders to receive transfusions. “Thousands of people living with sickle cell disease and thalassaemia will be eligible for this new world-first blood…

Comer Children’s Hospital at the University of Chicago will be one of the first centers in the U.S. to offer new gene therapies for sickle cell disease that were approved last year by the U.S. Food and Drug Administration (FDA). “I’m excited on behalf…

Most sickle cell disease (SCD) patients treated with the gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials were free from vaso-occlusive events (VOEs) after a median of three years of follow-up. That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American Society of Hematology…

Health regulators in the Kingdom of Bahrain have approved the gene-editing therapy Casgevy (exagamglogene autotemcel) to treat people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This decision makes Bahrain, a small island nation in the Persian Gulf, the second country to approve Casgevy. The therapy was…