John Leonard, Intellia CEO

With new up­date in hand, In­tel­lia CEO now claims one-time HAE gene edit­ing treat­ment 'may be a func­tion­al cure'

In Sep­tem­ber, In­tel­lia gave a first look in­to its one-time treat­ment for hered­i­tary an­gioede­ma — a dis­ease that caus­es se­vere swelling at­tacks — that di­rect­ly ed­its the gene un­der­ly­ing the pro­tein re­spon­si­ble for the at­tacks. It re­port­ed re­sults on three pa­tients: Two of the three were at­tack-free, and a third was at­tack-free af­ter 10 weeks.

At the time, In­tel­lia pres­i­dent and CEO John Leonard said he was “strong­ly en­cour­aged” by the da­ta read­out. But with the lat­est cut of da­ta, pre­sent­ed at an ACAAI meet­ing on Sat­ur­day, Leonard is ready to make a bold­er claim — that the treat­ment “may be a func­tion­al cure,” he told End­points News.

Endpoints News

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