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 de­fi­cien­cy.

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