We are very excited to provide updated clinical safety and efficacy data from 28 individuals with sickle cell disease treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2/3 RUBY clinical trial at the American Society of Hematology (ASH) annual congress. Participants were at a median (range) of 9.5 (0.7-25.2) months post-reni-cel infusion, with 11 having >1 year follow-up. Of the 28 participants, 27 were VOE-free post-reni-cel infusion. Early total hemoglobin normalization was observed, with mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 (n=18), as well as rapid and sustained improvements in fetal hemoglobin (HbF) to ≥40% and mean corpuscular HbF concentration per F-cells well above the anti-sickling threshold. Allelic editing levels in both peripheral blood nucleated cells and bone marrow–derived CD34+ cells were high, with mean editing levels of 75.1% (n=8) and 87.8% (n=7) at Month 12, respectively. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained over time. Finally, meaningful improvements in patient-reported outcome domains for pain, physical function, and social roles and activities were observed. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. If you have any questions or want more information, please reach out or check the links below! Poster can be found here - https://lnkd.in/eUSdVYyG More information on the Ruby Trial can be found here - https://lnkd.in/emSvRPqe Long-term follow up information can be found here - https://lnkd.in/ebuETUd7 #ASH24 #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #GeneEditing #GeneTherapy #Hematology #SickleCellDisease
#News: Editas Medicine reports updated clinical data from the RUBY trial of reni-cel in patients with severe sickle cell disease at the American Society of Hematology Annual Meeting. Read the press release for details: https://bit.ly/3ZzBhXk #ASH24 #geneediting #sicklecell #genetherapy