Shares of Intellia Therapeutics Inc. (NASDAQ: NTLA), a genome editing start-up, rose 33.7% in January, according to data from S&P Global Market Intelligence. It was a huge month for CRISPR-Cas9 related stocks generally -- for no particular reason -- and Intellia benefited.
So far, Intellia has shown it can get CRISPR/Cas9 genome-edited hematopoietic stem cells to boost fetal hemoglobin production by 80% to 95% when those cells are transplanted into mice, among other pre-clinical achievements. Unfortunately, the company still can't say when it will be ready to test a candidate derived from its drug-discovery platform in humans.
Roughly 1% of novel candidates in pre-clinical development ever make it to an FDA review, and just a fraction of those go on to become blockbuster drugs. Yet despite such long odds, Intellia's market cap is a head-scratching $956 million at the moment.
As is common with hot new biomedical technologies, stocks pegged to the underlying tech tend to make dramatic moves on any related news. So what might have been biggest reason for investors to get excited about Intellia and its peers last month didn't come from the biotechs themselves. Intellia, Editas Medicine (NASDAQ: EDIT), and Crispr Therapeutics (NASDAQ: CRSP) all started rising after The Wall Street Journal published a report that suggested Chinese investigators had begun human studies of experimental therapies developed using CRISPR-Cas9 gene-editing techniques years ago.
So far, Crispr Therapeutics is the first among its peers to give us a timeline for beginning human trials: The tentative front-runner in the niche intends to start them later this year in partnership with Vertex Pharmaceuticals. Intellia sports a couple high-profile partnerships as well.
Regeneron signed a 10-target deal with Intellia back in 2016 and wants to begin with a transthyretin-related amyloidosis (ATTR) candidate. But neither company has indicated when it expects to file an initial new drug application for their ATTR candidate, which the FDA must approve before they can begin clinical trials.
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