In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

The first in vi­vo CRISPR/Cas9 gene edit­ing pro­gram has some new dura­bil­i­ty da­ta show­ing sus­tained re­duc­tion of a tox­ic pro­tein in AT­TR amy­loi­do­sis at all four dose lev­els in a small 15-pa­tient study.

In­tel­lia Ther­a­peu­tics pre­sent­ed the much-an­tic­i­pat­ed da­ta for its Re­gen­eron-col­lab­o­rat­ed NT­LA-2001 Fri­day morn­ing, adding to the ini­tial Phase I re­sults it first de­liv­ered al­most a year ago to the day.

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