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Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
Their discovery may chart a path to a small-molecule drug that could be made available to all patients with sickle cell disease, as well as those with beta thalassemia. In sickle cell disease ...
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EDIT Inks Collaboration Deal to Develop Novel Gene Editing Therapies
VRTX obtained a non-exclusive license to utilize Editas’ Cas9 gene editing technology for ex vivo gene editing medicines ...