The latest biopharmaceutical company testing Wall Street’s belief in the promise of gene modification has gone public. Intellia Therapeutics (NASDAQ: NTLA) of Cambridge, MA, which owns rights to an important but disputed piece of the CRISPR-Cas9 gene editing technology, sold 6 million shares at $18 each this evening to raise $108 million. Its stock will trade on the Nasdaq starting tomorrow under the ticker “NTLA.”
The pricing is a 27 percent bump over Intellia’s goal last week of selling 5 million shares in the $16 to $18 range. It does not include another $55 million Intellia planned to sell separately to two drug-development partners.
Intellia joins Editas Medicine (NASDAQ: EDIT), also of Cambridge, Cellectis (NASDAQ: CLLS) of Paris, France, and Sangamo Biosciences (NASDAQ: SMGO) of Richmond, CA, as publicly traded biotechs using gene editing to develop their main products.
Only Sangamo has yet to test a potential therapy in human clinical trials. A fifth company, Crispr Therapeutics, is still private but has attracted nearly $100 million in venture investment. Crispr, Editas, and Intellia all claim rights to key CRISPR-Cas9 discoveries, either patented or in dispute.
The gene editing companies have formed partnerships with several drug makers, including Novartis (NYSE: NVS), Juno Therapeutics (NASDAQ: JUNO), Regeneron Pharmaceuticals (NASDAQ: REGN), Pfizer (NYSE: PFE), Biogen (NASDAQ: BIIB), Vertex Pharmaceuticals (NASDAQ: VRTX), and Bayer, for therapies that could eventually treat cancer, blood disease, cystic fibrosis, liver disease, and more. Intellia’s technology platform comes from the University of California, Berkeley biochemist Jennifer Doudna and her colleagues who first described the therapeutic potential of CRISPR-Cas9 in a 2012 paper in Science.
Doudna’s camp has been battling rivals from the Broad Institute of MIT and Harvard to establish patent rights in the field. The U.S. Patent and Trademark Office is now weighing the arguments side by side in a rare kind of hearing that could last months or years. Editas is the benefactor of the Broad’s patents, which the PTO granted in 2014 but agreed to reconsider in the face of the Doudna-Berkeley camp’s challenge.
Neither the patent controversy nor a flagging stock market has slowed the IPO march. Editas in February ended up selling more than half of its new shares to inside investors. Like Editas, Intellia has expected insiders to snap up a big portion of its shares. Before the deal, insiders aimed to buy IPO shares worth $30 million, according to Intellia regulatory filings. Regeneron and Novartis, Intellia’s two corporate partners, also pledged to buy $55 million in Intellia stock separately from the IPO, bumping Intellia’s gross haul to $163 million. (Intellia can add more to the haul in coming weeks; its bankers have the option to buy 900,000 more shares.)
Novartis has rights to use Intellia’s technology to develop treatments for certain cancers and blood disorders by extracting cells from patients, modifying them outside the body, and putting them back into the patients. Intellia is also pursuing treatments for liver diseases that can be injected directly into a patient’s body. In a deal announced last month, Regeneron now shares with Intellia the ownership of a program to develop a drug for the rare liver disease transthyretin amyloidosis. Intellia has reserved for itself the rights to other in vivo liver programs.
The CRISPR race to the clinic is on. Editas has said it aims to have its first program, for a rare form of blindness called Leber congenital amaurosis, in clinical trials by 2017. Intellia’s timelines remain more vague. In regulatory filings the company said it wanted to have in the next 12 to 24 months one or two liver-focused programs in “IND-enabling studies”—the final step before getting FDA’s green light for clinical trials. Intellia also noted that in 2018 Novartis could ask FDA permission to start a trial using CRISPR-modified blood stem cells as a treatment.