Gene therapies for hemophilia hit the mark in clinical trials

Although the first gene therapy proposals are almost 50 years old1, the past year has been a coming of age for this field. In March, after approval by the European Medicines Agency the previous year, the first patient was treated with a commercial gene therapy to correct an inherited immunodeficiency by modifying blood stem cells ex vivo. In August, a genetically modified cell product was approved for the first time by the US Food and Drug Administration (FDA), comprising engineered T cells for cancer immunotherapy. In November, a patient was treated for a genetic deficiency with in vivo genome editing for the first time…

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Fonte: nature medicine volume 24 | number 2 | february 2018