Exclusive: Jim Wilson startup gets FDA green light for first test of gene editing in babies

Nearly 25 years ago, a young man with an inherited liver disease died from a vicious immune reaction to an experimental gene therapy. Now Jim Wilson, the scientist behind that failed treatment, is back with a new attempt to cure the disease, this time by editing the genes of newborns and infants.

Philadelphia-based iEcure, one of several biotech companies spun out of Wilson’s expansive gene therapy program at the University of Pennsylvania, is preparing to treat babies up to 9 months of age with the severest form of the metabolic condition, called ornithine transcarbamylase deficiencyornithine transcarbamylase deficiency.

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来源: EndPoints

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