Intellia Therapeutics was one of the first biotech companies built around the Nobel prize-winning, gene editing technology known as CRISPR. But, while Intellia’s clinical progress has at times displayed CRISPR’s massive therapeutic potential, the company has burned through more than $2 billion since inception and twice laid off staff amid shifting research plans.
Its share price sits near record lows, too, following the death of a study participant and suspension of one of its top programs.
Intellia still has a shot at success, though. In the first half of 2026, it’s expecting results from a Phase 3 trial testing a possible treatment for a rare condition called hereditary angioedema. That therapy, named lonvo-z for short, has shown the potential to durably reduce the rates of the swelling attacks that characterize the condition.
Intellia has billed this therapy as a possible “functional cure” that might alleviate the need for the frequent injections or pills used to prevent or treat symptoms. It also, so far, hasn’t been beset by the same kind of liver-related issues as another program, nex-z, which is being developed for a type of genetic heart condition.
The results Intellia accrued so far suggest a “good probability of success” in the trial and, assuming an approval afterwards, a “competitive commercial profile,” wrote Evercore ISI analyst Jonathan Miller.
Intellia has pointed to important, technical differences between lonvo-z and nex-z — such as the genes they target and the RNA that “guides” them to it — that could lead to different outcomes. The HAE patients receiving lonvo-z are also typically younger and healthier than those getting nex-z.
“[W]e view lonvo-z and nex-z as absolutely distinct from each other and the patient experience thus far aligns with that,” said CEO John Leonard, on a November conference call, adding that the company expects lonvo-z to “redefine the HAE treatment landscape.”
Yet history isn’t on Intellia’s side. Genetic medicines have struggled to sell in areas where, as with HAE, multiple effective medicines exist. Because of those options, Intellia may need to prove exceptionally potent, durable and safe to entice patients and physicians to try it instead of existing options.
Miller, of Evercore, projects sales would top out at $500 million annually in peak years, given the way gene therapy launches have gone so far. — Ben Fidler
Link to trial
