Michael Jaskolka, PhD’s Post

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Senior Scientist I - Translational Science at Editas Medicine

We are excited to provide updated clinical safety and efficacy data from 7 transfusion-dependent beta thalassemia patients treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2 EdiThal clinical trial at European Hematology Association (EHA) annual congress. Treated patients had early and robust increases of total hemoglobin (Hb) and fetal hemoglobin, with total Hb rising to or above the transfusion independence threshold of 9.0 g/dL. All patients have been transfusion free for a range of 4.1 to 12.8 months after receiving the last red blood cell transfusion at 0.5–2.2 months post-reni-cel infusion. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. #BetaThalassemia #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #EHA #GeneEditing #Hematology

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