When it comes to picking winning biotech companies, sometimes it pays to go with players who are at the cutting edge of their field. On Oct. 18, regulators at the Food and Drug Administration (FDA) confirmed that Intellia Therapeutics (NASDAQ: NTLA) is a leader in gene editing: The FDA approved the company's application to proceed with its phase 3 clinical trial for its candidate NTLA-2001, which aims to treat transthyretin (ATTR) amyloidosis, a fatal protein misfolding disorder of the liver. The trial is anticipated to start before the end of this year, and when it does, it'll be the first-ever late-stage test of a therapy designed to treat or cure a hereditary condition by permanently editing patients' genomes.

But investing in pioneers carries additional risks, as there may be many potential pitfalls that so far remain unknown or poorly understood. So is Intellia worth an investment -- on the chance it makes an even bigger mark on history by commercializing one of its gene-editing therapies sometime in the coming years -- or is its foray into the unknown too uncertain to be investable?

The case for buying Intellia stock is inseparable from the success of its bid to commercialize NTLA-2001. While it has other gene-editing therapies in clinical trials in its pipeline, like its NTLA-2002 program to treat or cure hereditary angioedema (HAE), the ATTR program is the one that'll make a bigger splash if it succeeds -- because it stands to be the first curative gene-editing medicine on the market. On that note, Intellia's chances in the ATTR market may or may not be large enough to be everything that investors are hoping for.

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Source Fool.com