Shares of Intellia Tharapeutics Inc. (NASDAQ: NTLA), a start-up biotech with an eye on CRISPR-based gene editing, slipped 14.7% in December, according to data from S&P Global Market Intelligence. Data for the company's sickle-cell disease candidate wasn't robust enough to draw attention away from a peer's progress.
Last month, both Crispr Therapeutics Inc. (NASDAQ: CRSP) and Intellia Therapeutics announced pre-clinical data regarding their most advanced programs. Both aim to help sickle-cell disease patients produce enough fetal hemoglobin to reduce their dependence on frequent blood transfusions.
Before we make mountains out of molehills, it's important to remember that neither of these companies has given one of their new drug candidates to a human being. That said, Crispr Therapeutics showed its candidate was able to edit its target gene more than 90% of the time, which looks a bit better than a target-site modification rate of approximately 80% to 95%, which Intellia touted the following day.
The recent appointment of John Leonard, M.D., an industry veteran from AbbVie, shows Intellia is serious about moving a candidate into clinical-stage development, but the new CEO has a lot of catching up to do. The company's timeline for beginning clinical trials with a sickle-cell candidate is fuzzy, at best. Crispr Therapeutics, on the other hand, submitted an application to begin human studies with CTX001 to European regulators last month.
While investors wait for Intellia's sickle-cell program to catch-up, they'll want to keep an eye out for non-human primate data from a transthyretin amyloidosis candidate, in which it's partnering with Regeneron. If successful, the partners could have an application to begin clinical trials in front of the Food and Drug Administration in the first quarter of the year.
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