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…
Fresh from the biotech pipeline—2017
The mid-December approval of a gene therapy packaged inside an adeno-associated virus (AAV) vector to treat patients with certain inherited retinal diseases capped several decades of effort to bring in vivo gene therapy to patients. It was also the cherry on top of an impressive year… Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 2 FEBRUARY 2018 131…
FDA warns public of dangers of DIY gene therapy
People are experimenting on themselves with unlicensed gene therapies and publicizing the events widely, prompting the US Food and Drug Administration (FDA) to issue a statement on November 21 cautioning against do-it-yourself (DIY) gene therapies… Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 2 FEBRUARY 2018…
Biopharma deal-making in 2017
Drug makers in 2017 again faced many of the uncertainties and pressures experienced in 2016, including continued public and political criticism of drug pricing, and the urgency to put the patient and value-based health care at the forefront of medicine… Click here to original publication. Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 17 | FEBRUARY 2018 | 91…