Editas reports positive Phase 1/2 RUBY trial data for EDIT-301 in sickle cell disease

Editas Medicine, Inc.

First two patients engrafted successfully; no vaso-occlusive events after treatment with EDIT-301.
Patient 1 at 5 months post-treatment: total hemoglobin 16.4 g/dL, fetal hemoglobin 45.4%, HbF pancellular >95%.
EDIT-301 well-tolerated; safety profile consistent with myeloablative conditioning and autologous transplant.
First clinical use of proprietary AsCas12a nuclease to edit human cells; further data expected mid-2023.

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