A Communications Biology study functionally maps genes and noncoding loci controlling red-cell density, a phenotype relevant to hydration and hemoglobin concentration. The resource advances target discovery; its therapeutic direction is unresolved.
Investigators reported four recipients of autologous hematopoietic stem and progenitor cells edited ex vivo with tBE, a transformer base editor intended to reactivate fetal hemoglobin. One participant had sickle cell disease and three had…
A non-peer-reviewed study directly compared Cas9 ribonucleoprotein electroporation with lipid-nanoparticle delivery of Cas9 messenger RNA to disrupt the erythroid BCL11A enhancer in primary human hematopoietic stem and progenitor cells. Electroporation produced higher editing, while…
FDA expanded Casgevy to patients aged two years and older with sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia, lowering the prior age threshold from 12 years. The July 1 approval…
St. Jude researchers found that hematopoietic stem cells from children and young adults with sickle cell disease carry premature-senescence features and impaired blood-forming capacity. In xenograft models, senolytic treatment, including dasatinib plus quercetin, reduced…
Agios will not advance tebapivat in sickle cell disease after a 59-patient Phase 2 trial failed to establish a differentiated hemoglobin response. Response rates ranged from 29.4% to 47.1% across three doses versus 33.3%…
Agios Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease (SCD) and granted Priority Review, establishing a PDUFA target…
Beam Therapeutics announced updated biomarker data from the Phase 1/2 BEACON trial of ristoglogene autogetemcel (risto-cel), reinforcing its position as the leading base-edited cell therapy program in sickle cell disease ahead of a planned…
Agios Pharmaceuticals submitted a supplemental NDA to the FDA seeking accelerated approval of mitapivat for sickle cell disease, positioning the oral PKR activator in a highly competitive emerging therapeutic class.
