Sickle Cell Disease
The platform features incubation technology able to replicate cell microenvironments associated with SCD sickling.
Patients with follow-up of one month or longer saw hemolysis marker improvement and reported no vaso-occlusive crises.
In a phase III trial 90% of evaluable patients were free of severe vaso-occlusive crisis for at least 12 consecutive months.
Reni-cel led to hemoglobin normalization and increased fetal hemoglobin in patients with sickle cell disease.
An expert panel's review finds SCT has often been misattributed as the direct cause of mortality in patients who have SCT.
Preliminary efficacy and safety findings from a multicenter, open-label, phase Ib/II prospective study have been encouraging.
In a study SCT appeared linked to an increased risk of pregnancy-associated VTE, pulmonary embolism, and isolated DVT.
Patients receiving mitapivat showed a statistically significant hemoglobin response rate compared with placebo.
A review article describes current clinical knowledge of the role of inflammation and the activity of immune cells in SCD.
A study's efficacy and safety findings show promise for lovo-cel as a one-time intervention for SCD with history of stroke.
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