GMP-compliant Non-viral CRISPR-mediated Process Correcting the Sickle Cell Disease Mutation in SCD Patient CD34+ Cells Achieves 60% Wild Type Adult Hemoglobin Expression in Differentiated Erythrocytes.

The c.20A>T mutation of -globin gene causes sickle cell disease (SCD). Allogeneic hematopoietic stem cell (HSC) transplantation can cure SCD, but most lack a suitable donor. Ex vivo gene therapy strategies, including lentiviral mediated gene transfer or endonuclease mediated BCL11a knockdown allowing fetal hemoglobin (Hb) induction, are currently under evaluation.

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Fast Tracking Biotherapeutic Development via Flow Electroporation™ Technology for Large Scale Transient Expression and Cell Line Generation Using Your CHO Cell Line of Choice

Companies are turning to transient production of antibodies during early development to delay stable cell line generation, accelerate timelines, and reduce costs. A key factor for the success of this approach is the production of high quality antibodies with a high degree of similarity to stably produced proteins.

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