FDA al­lows first piv­otal tri­al of an in vi­vo gene edit­ing treat­ment from In­tel­lia

The FDA cleared In­tel­lia Ther­a­peu­tics to run a Phase III study of its CRISPR-based ther­a­py for transthyretin (AT­TR) amy­loi­do­sis with car­diomy­opa­thy, paving the way for the first piv­otal study of an in vi­vo gene edit­ing treat­ment in the US.

The gene edit­ing com­pa­ny said Wednes­day it ex­pects to start the Phase III study by the end of the year. The biotech, co-found­ed by No­bel lau­re­ate Jen­nifer Doud­na, has yet to run a study in the US. In­tel­lia en­rolled all 72 pa­tients in its ear­ly-stage tri­al of the AT­TR amy­loi­do­sis treat­ment in the UK, France, New Zealand and Swe­den, ac­cord­ing to the fed­er­al clin­i­cal tri­als data­base.

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