Terumo Blood and Cell Technologies is working to improve treatment for pregnant sickle cell sufferers, publishing first-of-its-kind international consensus recommendations for these patients.
In late 2023, Vertex Pharmaceuticals and CRISPR Therapeutics made history by gaining the first FDA approval for a CRISPR-based drug, exagamglogene autotemcel (Casgevy) for the treatment of sickle cell disease with vaso-occlusive crisis.
After voluntarily pausing a phase 1/2 trial due to safety concerns in January 2023, the gene edited cell therapy firm Graphite Bio has discontinued the development of nulabeglogene autogedtemcel (nula-cel), its lead candidate cell therapy for sickle cell...
Biopharmaceutical company Global Blood Therapeutics specializes in conditions that often disproportionately impact underrepresented communities, such as SCD.
The FDA has lifted the clinical holds on bluebird bio’s trials of gene therapies in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), an inherited blood disorder.
