A gene editing technology called CRISPR has been called “The Biggest Biotech Discovery of the Century” by the MIT Technology Review and Forbes magazine has said that this technology could change biotech forever. Some surprising news came out of China this week, when it was announced that Chinese scientists had utilized CRISPR to alter the DNA of human embryos. The experiment attempted to modify a gene in human embryos that causes a fatal blood disorder. This change would then go on to alter the DNA of every cell so any changes made could then be passed on from generation to generation. The ethical considerations immediately came front and center with talks of what guidelines should be used to control this technology. Is it okay to eradicate hereditary diseases but not okay to create designers babies? Regardless of the ethics of gene editing, it’s potential as a technology is incredibly powerful. There are three primary methods used to edit genes currently:
The “zinc finger” method is expensive and TALENS is said to be inefficient. It is CRISPR that has generated the most excitement up to this point, and right now it is a mad rush as companies look to claim their stake in this technology through patent filings. So who are some of the main players involved in this space right now?
Maybe the most promising candidate in the gene editing space so far is Editas, a company that we profiled before which was founded by 5 of the world’s leaders in gene editing. Backed by a $43 million first round of funding, Editas has exclusive rights to the one issued patent for CRISPR granted to the Broad Institute and Harvard University. Since then, Editas has nailed down key exclusive IP agreements with Massachusetts General Hospital, and Duke University for key CRISPR technologies.
Caribou Biosciences (private)
Backed by an $11 million funding round from Atlas Venture, Caribou was founded by Jennifer Doudna who was also one of the cofounders of Editas. When the key CRISPR patent was granted to another Editas founder, Feng Zhang, Jennifer broke off from Editas taking her intellectual property in the form of her own pending patent for CRISPR. It is with this patent that she is hoping to stake her claim.
Founded in 2014 by Caribou Biosciences and Atlas Venture, Intellia was funded by both Atlas Venture and Novartis Institutes for Biomedical Research (NIBR). Caribou has outlicensed their technology to Intellia for exclusive licensee for all human therapeutic applications Novartis has exclusive rights to use Intellia’s CRISPR platforms to develop CAR-T therapies. Novartis also receive non-exclusive rights for limited in vivo therapeutic applications of CRISPR systems and has a 5-year collaboration in place with Intellia for which they will provide an initial payment, technology access fees, and funding.
CRISPR Therapeutics (private)
CRISPR Therapeutics was founded just one year ago in April of 2014 by Emmanuelle Charpentier, also one of the co-inventors of the CRISPR/Cas9 technology. Emmanuelle Charpentier, who worked with Jennifer Doudna on CRISPR, took her stake in the same patent and co-founded CRISPR Therapeutics in Switzerland. CRISPR Therapeutics is backed by Versant with a $25 million first round of funding.
Cellectis is a $1.1 billion French company that is involved in both gene editing and cancer immunotherapy. The Company has worldwide rights to a patent family titled “Engineering Plant Genomes Using CRISPR/Cas Systems” upon which they have developed a platform to improve the quality of crops for the food and agriculture industries. Just last month, shares of Cellectis became available to U.S. investors through an ADR offering (NASDAQ:CLLS).
Precision Biosciences (private)
Precision’s Directed Nuclease Editor™ (DNE) technology enables the production of genome editing enzymes. Precision controls a growing patent estate consisting of over fifteen allowed genome engineering patents in the U.S., Europe and Australia. Precision has asserted twelve of these patents against Cellectis in the United States. Those lawsuits are currently pending in the United States District Court for the District of Delaware. They recently had a patent claim upheld when it was challenged by a third party.
California based Sangamo is a $1 billion company that uses a gene editing system based on zinc-finger nucleases and has quite a head start over Editas. Sangamo has entered Phase 1 clinical safety trials for their gene editing technique that is showing encouraging results as a possible functional cure for HIV.
While it seems like Editas has the lead here, nothing is certain until the dust settles. Even more importantly, nothing says that CRISPR is the technique to use going forward for gene editing or even that gene editing will become commercially viable. In the meantime, there are a few publicly traded companies above that may be able to provide retail investors some exposure to the exciting technology of gene editing.
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