Intellia suggests ATTR amyloidosis gene editing treatment has over a year of durability as it gears up for PhIII

In­tel­lia on Thurs­day pre­sent­ed new re­sults for its gene edit­ing treat­ment in 65 pa­tients with AT­TR amy­loi­do­sis — a con­di­tion in which mis­fold­ed pro­teins build up in the body — and the da­ta sug­gest that the treat­ment can low­er lev­els of the pro­tein in the blood by around 90%.

In 62 pa­tients who re­ceived a one-time dose of at least 0.3 mg/kg of NT­LA-2001, the me­di­an re­duc­tion in lev­els of transthyretin, the pro­tein that gets mis­fold­ed, was 91% af­ter one month. And in 29 pa­tients who were dosed at least one year ago, that re­duc­tion has held, ac­cord­ing to In­tel­lia.

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