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),…
Throughout 2023, Sickle Cell Disease News brought readers coverage of the latest clinical research and scientific breakthroughs related to sickle cell disease (SCD). Here are the year’s top 10 most-read articles, each with a brief description. We hope to remain a dependable resource for the SCD community in…
RoslinCT will manufacture the groundbreaking new gene therapy Casgevy (exagamglogene autotemcel) for sickle cell disease (SCD) patients in the U.S. and U.K. RoslinCT, a cell and gene therapy contract development and manufacturing company, has facilities in Edinburgh, Scotland and Hopkinton, Massachusetts. The company worked with Vertex…
On Dec. 8, the U.S. Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), a groundbreaking decision that now offers patients more than one potentially curative option that targets the underlying cause of the disease. Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel) — formerly…
A European Medicines Agency committee has issued a positive opinion on the gene-editing therapy Casgevy (exagamglogene autotemcel) in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The Committee for Medicinal Products for Human Use (CHMP) recommendation will be considered by the European Commission in making a final…
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