Slowly but surely, CRISPR-Cas9 gene editing treatments are working their way towards human clinical testing, spurring more large pharmaceutical companies to get in on their future. The latest today is Allergan, which has just aligned itself with Editas Medicine on treatments for a group of genetic eye diseases.
Allergan (NYSE: AGN) will pay Cambridge, MA-based Editas (NASDAQ: EDIT) $90 million in cash up front to get an option to license up to five experimental gene editing treatments for eye diseases—including Editas’s lead program, a drug for a rare form of genetic blindness called Leber Congenital Amaurosis type 10. Editas expects to file papers with the FDA by the end of the year to start the first human trials of the LCA10 treatment. The company’s pipeline also includes experimental therapies for a form of retinitis pigmentosa called Usher Syndrome, and Herpes Simplex Virus type 1, which can crop up in the eye.
The Editas didn’t disclose any other financial figures related to the transaction, just that it would get downstream payments if Allergan grabs rights to the eye drugs and they progress. Editas gets an option to share U.S. rights to two of the treatments in the alliance.
The deal is right in Allergan’s wheelhouse, adding more experimental eye drugs to the pipeline of a company that generated $660 million in revenue for eye therapies in 2016—mainly from treatments for glaucoma and dry eye disease. But the partnership is the latest mainstream stamp of validation for CRISPR-Cas9, a gene editing system that offers the potential to deliver a one-time fix for a variety of genetic diseases. CRISPR-Cas9 is a two-part system consisting of a pair of molecular scissors and a programmable guide that points the scissors to cut specific places in cell’s DNA. CRISPR’s potential is massive, but human testing of these therapies is only just beginning, and the road ahead is likely fraught with challenges. It took decades, for instance, for the first approved gene therapy—another newer drugmaking method—to get to market. A number of safety and delivery challenges had to be overcome.
Nonetheless, there are now three publicly traded companies—Editas, Intellia Therapeutics, and CRISPR Therapeutics—advancing CRISPR-Cas9 treatments toward human clinical testing either on their own or with deep-pocketed biopharma partners. Editas already has an alliance with Juno Therapeutics (NASDAQ: JUNO). Intellia has deals in place with Novartis and Regeneron Pharmaceuticals (NASDAQ: REGN). And CRISPR Therapeutics inked partnerships with Bayer and Vertex Pharmaceuticals.
Editas could be the first into human clinical testing. with its LCA10 treatment, but the firm also has preclinical programs for Duchenne muscular dystrophy, cystic fibrosis, sickle cell disease, and alpha-1 antitrypsin deficiency under development.
Editas got a boost in February when founders at the Broad Institute of MIT and Harvard won a patent fight against a group led by the University of California, Berkeley, over ownership of CRISPR-Cas9 (though as Xconomy explained here, the ruling left the door open for UC Berkeley to make future gains). Shares are up about 25 percent since the ruling, and climbed another 10 percent, about $2.40 apiece, to $27.30 a share in pre-market trading.
Photo by flickr user Michael Gil, via a Creative Commons license