Editas Medicine presented updated safety and efficacy data from the Phase 1/2/3 RUBY clinical trial of reni-cel in 28 patients with severe sickle cell disease at the American Society of Hematology (ASH) Annual Meeting on December 9, 2024. As of the October 29, 2024 data cutoff, reni-cel was well-tolerated, with a safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant.
Efficacy data showed that 27 of the 28 patients were free of vaso-occlusive events (VOEs) post-reni-cel infusion. Patients demonstrated early normalization of total hemoglobin, with a mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 for 18 patients.
Rapid and sustained improvements were observed in fetal hemoglobin (HbF) levels, reaching a mean of 48.1% at Month 6, well above the anti-sickling threshold. Clinically meaningful improvements were also reported in patient-reported outcome domains for pain, physical function, and social roles and activities.
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