Sickle Cell Disease
Researchers also measured in the study cohort a very high discontinuation rate for this agent.
The agent notably in preclinical studies has accomplished increased HbF without any evident cytotoxicity.
Gene therapy is reshaping sickle cell care, raising awareness and advancing treatments beyond the lab.
Reduced-intensity HSCT conditioning with lentiviral gene therapy decreased severe vaso-occlusive events by more than 80%.
Evidence was presented at EHA2025 that the agent improves RBC health and blood flow in patients.
Two cases of patients with SCD and history of delayed hemolytic transfusion reaction were presented at EHA2025.
The analysis was an assessment of 15-year follow-up data from the CLIMB study and was presented at EHA2025.
Vivien Sheehan, MD, explained how a novel assay can guide personalized therapeutic approaches for sickle cell disease.
Rilzabrutinib significantly decreased spleen weight and reduced inflammation in mice with homozygous sickle cell genotype.
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