Intellia’s late-stage win tees up market test case for in vivo CRISPR

The first Phase III data from an in vivo CRISPR gene therapy were among the week’s clinical highlights, as Intellia’s lonvo-z produced efficacy in hereditary angioedema that looked broadly comparable to leading prophylactic therapies. The question is whether the appeal of one-time dosing will outweigh concerns about permanent in vivo gene editing.

Intellia Therapeutics Inc. (NASDAQ:NTLA) said lonvoguran ziclumeran, or lonvo-z, met the primary endpoint the Phase III HAELO study, reducing swelling attacks 87% versus placebo at six months. Mean monthly attack rate was 0.26 vs. 2.1 for placebo (p<0.0001)...