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TALEN, a gene-editing technology being used by the clinical-stage biopharmaceutical company Cellectis to develop potentially life-changing therapies, was able to correct the mutation that causes sickle cell disease (SCD) in patient cells. That’s according to a new study published by researchers from the company, along with colleagues from…

All 18 adults with severe sickle cell disease (SCD) treated with the gene-editing therapy renizgamglogene autogedtemcel — known as reni-cel — remained free of vaso-occlusive events, or VOEs, for up to nearly two years, according to new interim data from the Phase 1/2/3 RUBY clinical trial. The therapy, being…

Nearly all people with sickle cell disease (SCD) treated with the U.S.-approved gene-editing therapy Casgevy (exagamglogene autotemcel) were free of painful vaso-occlusive crises (VOCs) for at least one year, according to new data from a Phase 2/3 clinical trial. These updated results were presented at the annual…

The Sickle Cell Disease Foundation (SCDF), in partnership with the Center for Inherited Blood Disorders (CIBD) and The Hills Tandem, is leading a real-world health data initiative that is progressing toward its goal of improving sickle cell disease (SCD) patients’ life quality and transforming their care. The…

Legislation to provide new outreach and support services to Colorado residents living with sickle cell disease (SCD) is now law. Gov. Jared Polis (D) signed the measure (SB24-042) calling for the appropriation of $200,000 to make the Arie P. Taylor Sickle Cell Disease Outreach Program part of the…

Note: This story was updated June 17, 2024, to correct that “Shine the Light on Sickle Cell” is a SiNERGe campaign independent of the Sickle Cell Disease Association of America. The theme of this year’s World Sickle Cell Day is “Hope Through Progress: Advancing Care Globally” and supporters are all…

A second clinical study to test how well BioLineRx’s motixafortide helps mobilize hematopoietic stem cells for use in gene therapies in sickle cell disease (SCD) is expected to begin enrolling in the next few months. The planned Phase 1 study, called SCDSTEMM (NCT06442761), will run as a…

The Bahrain Oncology Center now is among the first in the world to offer Casgevy (exagamglogene autotemcel), an approved gene-editing therapy, to people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), a related blood disorder. That milestone, announced in a release by the country’s regulatory authority,…

Longer gaps in transitioning from pediatric to adult care were associated with more hospital stays for people with sickle cell disease (SCD), a study indicates. Young adults who had a delay of more than six months were twice as likely to be hospitalized over those who made the switch…

Certain blood biomarkers of fat dysregulation, inflammation, and red blood cell destruction, known as hemolysis, may be used to predict the short-term risk of acute chest syndrome (ACS) in people with sickle cell disease (SCD). That’s according to a new study from France, in which scientists identified these…