The Foundation for Sickle Cell Disease Research will evaluate VAS-101 in a phase 1 clinical study. Malignant neoplasm-associated hemophagocytic lymphohistiocytosis, COVID-19, and CAR T-cell therapy can each initiate CS. In a phase 1 trial the product performed favorably in aplastic anemia, myelofibrosis, and hypoplastic myelodysplasia. The reduced-dose oral regimen also had lower incidence of clinically relevant bleeding complications and mortality rate. The platform features incubation technology able to replicate cell microenvironments associated with SCD sickling. The FDA granted orphan drug designation to rilzabrutinib for warm autoimmune hemolytic anemia and IgG4-related disease. ASH Awards Commitee Chair Ivan Maillard, MD, PhD, and prior awardee Rinku Majumder, PhD, tell of the Grant's positive impact. Bexobrutideg, an orally bioavailable, brain-penetrant BTK degrader, is being evaluated in a phase 1a/b study. The agent recalibrates the immune system to attack tumors and increases cancer susceptibility to standard of care therapy. A phase 3 study comparing the treatments in multiple hematologic malignancies also saw greater overall survival at one year. As the most common bleeding disorder, von Willebrand disease is found in up to 1% of the United States population. Azercabtagene zapreleucel is an allogeneic CD-19-targeting therapy combining lymphodepletion chemotherapy with interleukin-2. The hepcidin mimetic increased hematocrit control and reduced both phlebotomy need and symptoms in a phase 2 trial. PGK1 antibody levels in patients with recovered immuno-related pancytopenia were negatively correlated with platelet levels. Thrombosis history was not predictive of overall survival in patients with pulmonary hypertension risk and PV, a study found. The oral agent exhibited promising hemoglobin response performance and safety in this stage of an ongoing phase 2/3 study. The first-in-class agent operates by increasing both von Willebrand factor and factor VIII levels in patients. Immune thrombocytopenia featured most prominently at the Meeting but new data was presented for several forms of the disease. Imetelstat is indicated for patients with lower-risk MDS who had an unsatisfactory response to or are ineligible to ESAs. In findings arterial thrombosis carried worse overall survival than venous thrombosis in this patient population.