Intellia Therapeutics Inc. got regulators’ green light to start US trials of its gene-editing treatment for a rare swelling disorder, a major step forward for the biotechnology company that sent the shares climbing the most in two months.
(Bloomberg) — Intellia Therapeutics Inc. got regulators’ green light to start US trials of its gene-editing treatment for a rare swelling disorder, a major step forward for the biotechnology company that sent the shares climbing the most in two months.
The Food and Drug Administration cleared Intellia’s request to start human trials of its Crispr therapy for hereditary angioedema, according to a statement Thursday. The step is largely seen as procedural, but it’s becoming less of a guarantee and a bigger milestone for novel drugs like Intellia’s that aim to make permanent changes to a person’s DNA.
The Cambridge, Massachusetts based company’s shares rose as much as 11% as of 10:50 a.m. in New York, the most intraday since Jan. 4.
The FDA last year put on hold an application from Verve Therapeutics Inc. over questions around whether the company’s gene-editing treatment for a different disease could result in unintended changes to DNA that could be passed on to offspring. The hold on Verve, which hit shares of Intellia and other gene-editing companies at the time, remains in place, the company said Thursday in a separate statement. Verve shares rose 4% in New York.
Unlike other technologies that deliver the gene-editing Crispr tool outside the body, Intellia’s experimental NTLA-2002 works inside patients. The company has been conducting its clinical trials in countries outside the US, including New Zealand, the Netherlands and the UK.
“This marks a milestone for the company and opens the doors for global registration trial of NTLA-2002,” Truist Securities analyst Joon Lee wrote in a note to clients. More importantly, this clears the stage for another regulatory application from Intellia later this year for a drug to treat a disease that afflicts many more patients, Lee said. Called transthyretin amyloidosis, the disease can result in nerve and heart damage.
That application for testing in that disease is more complex than the one that was just cleared, Intellia Chief Executive Officer John Leonard said in an interview. Intellia is seeking permission for a pivotal trial whose results would be used to pursue approval, and hashing out those details may take longer than the 30 days the FDA has to complete its review.
“I’m not saying that that’s not going to happen, but it’s a lot to do,” Leonard said.
(Updates with CEO comments starting in seventh paragraph)
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