CRISPR gene editing technology is hot, and AstraZeneca ($AZN) wants in on it. In a broad-ranging slate of tech collaborations, the pharma giant is allying itself with several top research institutions in the U.K. and U.S. to start honing its ability to apply gene editing techniques to its drug development work. And AstraZeneca used this alliance to pick some of the best brains in the business, without turning to any of the pioneers who have recently spawned a lineup of upstart biotechs.
Gene editing has been in the works for years, but a group of scientists has recently been lionized for developing the CRISPR-Cas9 approach to performing careful DNA surgery inside the nucleus of cells, which promises to have a profound impact on disease. When Novartis ($NVS) decided to tackle the research field, the Big Pharma first helped launch a biotech with Atlas Venture--Intellia--and then executed a commanding research collaboration aimed at moving programs into the clinic. AstraZeneca, though, is doing something far more complex; assembling a global team that can each apply expertise in working with the pharma giant's in-house experts. And they have the company's full set of core disease focuses in mind.
- The Wellcome Trust Sanger Institute in Cambridge, U.K., where AstraZeneca is building a big new HQ and research campus, will focus on switching genes "off" for cancer, cardiovascular, metabolic, respiratory, autoimmune & inflammatory diseases and regenerative medicine.
- The Innovative Genomics Initiative, a joint venture between the University of California, Berkeley and University of California, San Francisco, will focus on the role that inhibiting and activating genes has on disease pathology for the full lineup of disease categories.
- The big lab tech group Thermo Fisher Scientific will help with RNA-guided libraries to screen for new disease targets.
- The famous Broad Institute/Whitehead Institute in Cambridge, MA, will specifically focus on cancer cell lines in search of new oncology drug targets.
AstraZeneca has focused heavily on these kinds of major scientific alliances with top institutes to provide it with an ambitious discovery effort as it tries to create a new foundation for its once widely ridiculed R&D operations. That tie-in with academia has provided some world-class scientific credibility, but it also will eventually offer some insight into whether a big corporation can effectively pursue the kind of complex, innovative scientific teamwork needed to actually deliver new programs for clinical development. And it will test its model against Novartis's plan to tie in with a scientific pioneer, an approach that has delivered solid results so far with its CAR-T effort at the University of Pennsylvania.
Intellia, which in-licensed the tech from Caribou, is just one of several new CRISPR-Cas9 players. Editas was one of the first to get started, along with CRISPR Therapeutics in Switzerland. And the division of scientific players among the startups has created some clear tension over who owns the intellectual property involved in this new tech.
In AstraZeneca's defense, CEO Pascal Soriot has executed a blizzard of deals and restructurings in a frenzied effort to refocus and resurrect a once great R&D group. And the company has achieved some recognition for its come-from-behind effort to gain a second-tier spot in the race for immuno-oncology drugs. Only time will tell if Soriot's approach to scientific innovation is productive or not.
- here's the release