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…
A biomedical engineer at Kennesaw State University has been given a $426,000 grant to build a low-cost device helping to predict the risk of a stroke in children with sickle cell disease by using light to monitor blood flow to the brain. Paul Lee, PhD, an assistant professor at…
Ferriprox (deferiprone) showed comparable efficacy to Desferal (deferoxamine mesylate) in reducing blood transfusion-induced iron overload in children and adolescents with sickle cell disease (SCD) or other anemias, a recent study reported. Ferriprox is an oral treatment, while Desferal is administered via a subcutaneous (under-the-skin) infusion. As such, Ferriprox is…
The first patient has been dosed in BEACON, a Phase 1/2 clinical trial of a transplant-based gene-editing stem cell therapy called BEAM-101 that Beam Therapeutics is developing for sickle cell disease (SCD). After the transplant, stem cells began to “engraft,” meaning they started to grow and give rise…
Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older. The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also…
A $3-million federal grant to investigate the potential of repurposing a blood pressure medication for sickle cell disease (SCD)-related cardiac disease has been awarded to researchers at Indiana University School of Medicine. The grant from the U.S. Department of Defense was given to the study’s lead researcher Ankit A.
A month after its U.S. approval, Lyfgenia (lovotibeglogene autotemcel), Bluebird Bio’s gene therapy for sickle cell disease (SCD), is readying to treat patients there. According to the company, 35 out of 48 qualified treatment centers across the U.S. started accepting patient referrals as of Jan. 5. Treatment…
A small proof-of-concept clinical trial evaluating motixafortide for stem cell mobilization — regimens essential for gene therapies — in sickle cell disease (SCD) has dosed its first patient. Stem cell mobilization regimens currently available can cause serious side effects in SCD patients. This Phase 1 study (NCT05618301),…
Get regular updates to your inbox.