SAN FRANCISCO — The drug maker GlaxoSmithKline announced Thursday that it would team up with some of the nation’s most prominent CRISPR researchers to use the gene-editing technology in a search for new medicines, establishing a new lab in San Francisco and spending up to $67 million over five years.
Jennifer Doudna, the University of California, Berkeley, researcher who co-invented the CRISPR enzyme technology, will help lead the effort, along with Jonathan Weissman, a UC San Francisco researcher who has been using CRISPR to understand the function of individual human genes and how they work together. Both are Howard Hughes Medical Institute investigators.
The lab will be called the Laboratory for Genomic Research and will be based near UCSF’s Mission Bay campus. The money will fund 24 full-time University of California employees, in addition to as many as 14 full-time GSK employees. GSK’s machine learning and artificial intelligence groups will create computer systems that can handle the large amounts of data the project is expected to create. It will focus on immunology, oncology, and neurology.
“I think for all of us, we see a really kind of unprecedented opportunity to leverage the best of academic science and research here in the Bay Area with also the very best of pharmaceutical science to do things that in the past would’ve been either difficult or impossible to achieve,” Doudna said.
The effort got its start a little more than a year ago when Doudna had dinner at Garibaldi’s, a stylish California-Mediterranean restaurant near the UCSF campus, with Hal Barron, who had just started as GlaxoSmithKline’s chief scientific officer.
The topic: using CRISPR to transform the way that drug targets — the genetic or protein leads that researchers use as starting points for creating new medicines — are identified. It was a “wide-ranging” conversation, Doudna said, about fundamental science but also how that science could seed a drug pipeline that would be “very transformative” if they could get the balance between academia and industry right. Immediately, Doudna said, she introduced Barron to Weissman, who had been using CRISPR to alter genes in individual cells and, by doing so, figure out what they do.
“It was wonderful because it was an opportunity to take some of the tools that we’ve been building, and to be able to apply them to have an impact and help develop drugs,” Weissman said. Academic labs, he said, advance knowledge and train new scientists. But there can be frustration when it comes to translating those ideas into the creation of new medicines, which usually requires the involvement of private-sector companies.
Barron, Doudna, and Weissman will discuss the new laboratory at a STAT Plus event in San Francisco on Thursday night.
It’s important to remember that most of the time, when a genetic alteration seems related to a disease or some other physical change, researchers don’t know why, what it does, or even what gene is involved. It may not be part of the relevant gene, for instance — it could just be next to it.
Weissman’s work focuses on how CRISPR can change this in a way that was completely impossible before. The technology allows individual changes to be made in DNA in cells, and to see how they change. GSK’s Barron foresees pairing this information with machine learning to create giant interaction maps that will tell pharmaceutical companies where to direct their resources. “It’s fundamentally a belief, a strongly held belief, that technology really does drive innovation,” said Barron.
Barron also said he hoped the collaboration would create a middle ground in how GSK works with researchers. Right now, relationships tend to be based on one of two models: Either the company funds academics, or academics create a company. This laboratory will represent a somewhat different model.
That could also create challenges in managing the resulting intellectual property. The researchers said that all technologies developed by the laboratory will be made public, as will many biological discoveries. But many of the best drug targets will be licensed exclusively to GSK. Getting those details right has taken months. But Barron said that from the moment they had the discussion, the three of them were certain the new laboratory was going to happen.
“It’s going to be a phenomenal thing,” Barron said. “There was incredible excitement and I think full support and enthusiasm about putting this together, I don’t know, maybe even within minutes of discussing it. I’ve known Jonathan for 25 years. When Jennifer and I discussed it, it just became so clear.”
By Matthew Herper