For Intellia Therapeutics, CRISPR drug’s safety concern is an existential threat

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

Intellia Therapeutics will find it challenging to survive now that its CRISPR gene-editing treatment has been tied to severe, potentially fatal, liver toxicity. 

It is now difficult to imagine a viable future for the biotech company. The potential benefits of a one-time, potentially curative gene-editing treatment disappear when the very real possibility of dying from liver injury can’t be ruled out. 

A safety risk might be navigated if Intellia were pursuing catastrophic, fatal diseases without treatment options. It’s not. Both of the company’s lead programs are in diseases — transthyretin amyloidosis (ATTR) and hereditary angioedema (HAE) — in which recently approved drugs have proven to be highly effective and more convenient options are under development.

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