The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
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MaxCyte: Building the Future of Cell and Gene Therapy InnovationMaxCyte's technology ensures that a large number of T cells are successfully edited with CRISPR/Cas-9 molecular scissors, which is critical for the effectiveness of the therapy. Casgevy enables CRISPR ...
Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
Crispr Therapeutics AG has revenue declines but holds strong financials. Promising Sickle Cell therapy & 2025 data readouts ...
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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug"The editing therapy is a wonderful achievement ... It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production after birth. This lets fetal hemoglobin ...
The therapy, known as CTX001, is applied to blood stem cells removed from the patient to cleave the BCL11A gene that represses the production of fetal hemoglobin. These cells are then reinfused to ...
which is critical for the effectiveness of the therapy. Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient ...
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