Editas will highlight the P-I/II/III (RUBY) study data assessing the safety & efficacy of reni-cel to treat severe sickle cell disease at EHA 2024
In the study, all patients (n=18) were free of vaso-occlusive events (VOEs) at 2.4-22.8mos. follow-ups, mean total Hb was 14.3 g/dL (SD 2.1, n=9) & mean HbF was 48.5% (SD 3.7, n=10) at 6mos. when follow-up was carried out for ≥6mos. and mean percentage of F-cells exceeding 90% at 4mos. in all of them (n=12) with ≥4mos. follow-up
Furthermore, MCH-F of HbF-containing red cells (F-cells) remained >10pg/F-cell at 3mos. in all of them (n=14) with a follow-up of ≥3mos. and all patients experienced editing in the HBG1 & HBG2 promoter regions with improvement in hemolysis markers
Ref: Editas Medicine | Image: Editas Medicine
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