Sickle Cell Disease
Compared with placebo, crizanlizumab has superior safety and comparable efficacy in patients with sickle cell disease.
The study led by Paul George, MD, PhD, also highlights the importance of patient regimen adherence to secure these benefits.
The findings coincide with patient reports of experiencing increased vaso-occlusive episodes during menses.
The Foundation for Sickle Cell Disease Research will evaluate VAS-101 in a phase 1 clinical study.
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.
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