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ASH 2025 | Reni-cel in severe SCD: updated results from the RUBY trial
Rabi Hanna, MD, Cleveland Clinic, Cleveland, OH, shares updated results from the RUBY trial (NCT04853576), which is investigating renizgamglogene autogedtemcel (reni-cel), an investigational gene-edited autologous hematopoietic stem cell therapy, in patients with severe sickle cell disease (SCD). Dr Hanna notes that the safety and efficacy of this therapy are promising, with patients demonstrating sustained normalization of total hemoglobin, increased fetal hemoglobin, significant reductions in pain scores, and improvements in physical activity. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
I’m very excited to share at ASH 2025 the updated results from the RUBY study, which uses reni-cel, which is a gene editing treatment for patients with severe sickle cell disease. The study today will update the results of 40 patients who were enrolled, aged 12 to 50. And we now have really a more prolonged follow-up with a median of follow-up close to 13.6. And the main message that it is really showing consistently the same safety profile that we expect with a busulfan myeloablative...
I’m very excited to share at ASH 2025 the updated results from the RUBY study, which uses reni-cel, which is a gene editing treatment for patients with severe sickle cell disease. The study today will update the results of 40 patients who were enrolled, aged 12 to 50. And we now have really a more prolonged follow-up with a median of follow-up close to 13.6. And the main message that it is really showing consistently the same safety profile that we expect with a busulfan myeloablative. But importantly, it’s really fantastic efficacy. 38, I’m repeating, 38 out of the 40 patients are pain-free as of the last cutoff of the data. So this has shown us continued improvement for these patients. And importantly, today, we are also reporting the pain scale and also the physical activity. Both measurements of patient-reported outcomes are seeing continued improvement and normalization, almost to what is reported by the patient. So it’s not only that they are pain-free, but also their physical stamina, their pain score is back to what’s expected for the normal population.
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