Investors hoping to buy into the vast, yet still unproven drugmaking potential of the gene editing system CRISPR-Cas9 will soon have three different companies to choose from. CRISPR Therapeutics filed for an IPO on Friday, meaning it could soon join Boston-area rivals Editas Medicine and Intellia Therapeutics in the expanding club of publicly traded CRISPR-Cas9 drug developers.
Basel, Switzerland- and Cambridge, MA-based CRISPR has been slowly amassing rounds of venture funding, adding partners, and filling out an executive team over the past few years. Editas (NASDAQ: EDIT) and Intellia (NASDAQ: NTLA) have each gone public in 2016, and CRISPR has long been expected to join them. Should CRISPR complete the IPO, it’ll trade on the Nasdaq under the ticker symbol “CRSP.”
Over the past several years, CRISPR-Cas9 has captured the imaginations of scientists and drugmakers alike. It’s a tool currently used in research labs across the globe that one day might help perform genetic surgery to correct devastating diseases. There’s a long way to go first, of course. CRISPR-based therapies have only just begun their first clinical tests in human patients, and many questions about their safety and effectiveness will have to be answered. A high-profile patent fight between various of the technology’s scientific founders is also playing out as Editas, Intellia, and CRISPR push their way forward.
But CRISPR companies are also taking cues from the developers of gene therapies and RNA interference, two other complicated drugmaking methods that have gone through a series of ups and downs over the past few decades. Methods that gene therapy and RNAi drugmakers use to deliver their therapies into patients are a starting point for CRISPR drugs, before developers go on to potentially tackle more complicated biological problems.
Editas, for instance, is starting out developing a CRISPR drug for a form of genetic blindness—a different variation of the disease Spark Therapeutics (NASDAQ: ONCE) is going after with a gene therapy that could soon be approved in the U.S. Intellia has an experimental CRISPR drug for the liver disease transthyretin amyloidosis, the same disease targeted by Alnylam Pharmaceuticals’s (NASDAQ: ALNY) lead drug patisiran. And CRISPR is going after the blood diseases beta-thalassemia and sickle cell disease, each of which is the target of a gene therapy from Bluebird Bio (NASDAQ: BLUE).
Each of Editas, Intellia, and CRISPR was backed by different venture firms and formed partnerships with different drugmakers. Editas was formed by Third Rock Ventures, Flagship Ventures, and Polaris Partners, and has an alliance with Juno Therapeutics (NASDAQ: JUNO). Intellia, formed in part by Atlas Venture, has deals in place with Novartis and Regeneron Pharmaceuticals (NASDAQ: REGN). And CRISPR Therapeutics, started by Versant Ventures, inked big partnerships with Bayer and Vertex Pharmaceuticals (NASDAQ: VRTX). CRISPR formed a joint venture with Bayer called Casebia Therapeutics to develop gene editing drugs for cardiovascular diseases. The Bayer and Vertex partnerships could give CRISPR over $400 million in non-dilutive cash to work with, assuming it hits a variety of milestones.
CRISPR is developing both in vivo and ex vivo CRISPR therapies, meaning that some treatments will be infused directly into the body, and others will involve harvesting cells from a patient, modifying them in a lab, and returning them to the body. Like its rivals, CRISPR doesn’t have any drugs in clinical testing as of yet—it doesn’t expect to file papers with the FDA to begin its first trial until the end of next year. It is developing drugs for cystic fibrosis, sickle cell, and beta thalassemia, with Vertex. CRISPR also lists experimental treatments for hemophilia, severe combined immunodeficiency (an immune disorder known as “bubble boy disease”), glycogen storage disease 1a, cancer, and Duchenne muscular dystrophy in its pipeline. The blood disease drugs are expected to be CRISPR’s first in clinical trials.
CRISPR was initially formed as Inception Genomics in October 2013. Versant is CRISPR’s largest shareholder, with a 20.74 percent stake. Others include affiliates of Celgene (12.40 percent), Bayer (8.01 percent), and Vertex (7.6 percent), and venture firms SR One (9.69 percent), New Enterprise Associates (9.69 percent), and Abingworth (7.82 percent). The company has raised $293 million in equity financing, partnership payments and other financings since inception. It had about $247 million on hand at the end of June and has burned through about $60 million since inception. Bayer has agreed to buy $35 million in CRISPR stock in a separate deal at the IPO price, according to the IPO prospectus.