Gene Editing Phenom CRISPR Files for $90 Million IPO

Published: Sep 12, 2016

Gene Editing Phenom CRISPR Files for $90 Million IPO September 12, 2016
By Mark Terry, BioSpace.com Breaking News Staff

Cambridge, Massachusetts-based CRISPR Therapeutics filed with the U.S. Securities and Exchange Commission (SEC) on Friday, indicating that it plans a $90 million initial public offering (IPO). As yet, the date of the IPO has not been announced.

Headquartered in Switzerland, with a large facility in Cambridge, CRISPR was cofounded by Emmanuelle Charpentier, who discovered CRISPR in 2012 with Jennifer Doudna of the University of California, Berkeley. The company focuses on translating CRISPR-Cas9, genome-editing technology, into therapeutics for diseases. Cas9 is an enzyme that can be programmed with RNA to cut DNA at specific, targeted locations within the genome, which allows for easier, more specific gene editing.

CRISPR Therapeutics and CRISPR technology has been tangled up in lawsuits pretty much from the beginning and aren’t likely to be wrapped up any time soon. Doudna and Charpentier, who co-founded CRISPR Therapeutics with Rodger Novak, published the first paper describing the technology. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, filed a broad U.S. patent claim on the technology.

The technology was first described in the journal Science in 2012 by Doudna, then a biologist at the University of California, and Charpentier, then at the Max F Perutz Laboratories at the University of Vienna in Austria. However, Zhang, at the Broad Institute, first won a patent for the technology after submitting laboratory notes to prove he was the first inventor.

Doudna originally founded a company, Caribou Biosciences, but later cofounded Editas with Zhang and George Church, a professor at Harvard and MIT.

Other companies developing CRISPR include Editas Medicine (EDIT) and Intellia Therapeutics, both in Cambridge, Mass. Both Editas and Intellia have also launched IPOs. Intellia’s IPO raised $124 million in its debut in May. Editas raised $109 million in its IPO in February.

The Boston Business Journal, writes, “However, it faces challenges: Namely, for all the hype surrounding gene editing, neither of the other two companies in the field have seen much in the way of stock gains. Shares of Editas, the company that raised $94 million in February, have only gained three percent in the past seven months from the IPO price, while those of Intellia, which raised $163 (million) in the state’s largest recent biotech IPO this past May, have increased 17 percent in four months.”

CRISPR expects to sell its shares on NASDAQ under the ticker CRSP. The SEC filing also indicates that it is focused on early-stage treatments for hemophilia, Hulder syndrome, and severe combined immunodeficiency. Earlier reports showed it was focusing on sickle cell disease, Duchenne muscular dystrophy, and beta-thalassemia, with plans to study the technology in cancer.

CRISPR recently started a joint venture with Germany’s Bayer , called Casebia Therapeutics. It will be based in Cambridge, and is focused on developing and commercializing new drugs for blood disorders, blindness, and congenital heart disease using CRISPR’s gene-editing technology and Bayer’s protein engineering expertise and marketing skills. Bayer invested $335 million in the joint venture.

Although CRISPR-Cas9 does appear to be revolutionary as a technology tool, its products will face all the same competition in the marketplace as any other drug development process. STAT also notes that the SEC filing, “included the usual caveats about how its technology might never work, might never receive approval from the Food and Drug Administration, and might never make money. It also disclosed that developing CRISPR for therapeutic uses is a good way to lose piles of money, at least initially: The company reported an operating loss of $25.7 million in 2015 and that pace has only accelerated. It lost another $28.2 million in the first half of this year.

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