In­tel­lia presents a promis­ing snap­shot of its first da­ta for once-and-done in vi­vo CRISPR treat­ment for HAE

The first slice of da­ta has ar­rived from a dose es­ca­lat­ing study of In­tel­lia’s in vi­vo CRISPR treat­ment for hered­i­tary an­gioede­ma, and it’s a promis­ing first step to­ward a po­ten­tial once-and-done ther­a­py.

The CRISPR crew at In­tel­lia $NT­LA re­port­ed Fri­day morn­ing that six HAE pa­tients treat­ed with ei­ther the low 25 mg dose or the high 75 mg dose of NT­LA-2002 ex­pe­ri­enced a 65% and 92% mean re­duc­tion in plas­ma kallikrein. The low-dose group ex­pe­ri­enced a 91% re­duc­tion in HAE at­tacks af­ter the drug was used to knock out the KLKB1 gene in liv­er cells.

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