Gene-editing pioneers based at the University of California (UC) are partnering with GlaxoSmithKline (GSK) to establish a new laboratory that will explore how gene mutations cause disease, and develop new CRISPR-based technologies aimed at speeding up drug discovery.

Jennifer Doudna, Ph.D., of UC Berkeley, and Jonathan Weissman, Ph.D., of UC San Francisco (UCSF), are joining GSK’s CSO and President, R&D, Hal Barron, M.D., to launch the Laboratory for Genomics Research (LGR), under a five-year collaboration signed by UC and GSK.

Over that period, GSK has agreed to fund the LGR with $67 million toward facilities for 24 full-time university employees to be funded by GSK, as well as up to 14 full-time employees of the pharma giant.

Doudna and Weissman—who are also investigators with the Howard Hughes Medical Institute—will lead LGR as it focuses on immunology, oncology and neuroscience. The lab will be based in San Francisco on Illinois Street, near UCSF’s Mission Bay campus.

GSK said its artificial intelligence (AI) and machine learning group will help create the computational tools needed to analyze the data to be generated by LGR, with the goal of automating existing CRISPR approaches so that the analysis can be done at scale.

By applying AI and machine learning to identify correlations between genetic variants and disease, GSK and UC reason, their researchers can select for clinical trials those patients who are most likely to benefit, thereby speeding up the development of new therapies.

According to GSK and UC, the new LGR will bring together industrial and academic researchers under a single roof working on projects both together and independently, in what the company called a novel hybrid model. LGR’s research projects will be focused on technologies, new drug targets, and biological mechanisms intended to foster both academic and industrial advances.

Creative Science, Robust Technology

“LGR is about building that space where creative science is partnered with the development of robust technology that will help develop tomorrow’s drugs,” Doudna said in a statement. “I think we’re going to be able to do science that none of us can even imagine today.”

LGR will also be a resource for investigators at UC Berkeley and UCSF, who will be able to access and use its technology to answer their own biomedical or other biological questions, as well as develop new tools that explore how genes work.

“One of our key goals is to advance the field overall and make these tools as broadly available as possible. The LGR screening center will enable labs at UCSF and Berkeley, and having access to it will give our scientists opportunities to advance their research in ways that would be very hard for them to do in their own labs,” Weissman stated.

Weissman’s lab has developed a modified version of CRISPR technology, CRISPRa, which was shown last year to reverse genetic forms of severe obesity in two different mouse models, without genome editing. CRISPRa uses the CRISPR guidance system to target a particular gene sequence, but instead of using Cas enzymes as molecular scissors to cut out and repair or replace sequences, the technology amplifies existing gene activity to ramp up protein production

At LGR, tools that are developed there will be described in published papers, subject to intellectual property provisions, and will be available for use by other academic and non-profit labs, GSK and UC said.

A Joint Steering Committee with equitable representation by UC and GSK representatives will oversee the partners’ collaboration, with additional joint sub-committees covering patents, scientific management, and project management.  Doudna and Weissman will be members of the Joint Steering Committee, together with GSK’s new head of functional genomics, Chris Miller, Ph.D., who recently joined the company from AbbVie.

LGR will build on the work of:

• UC’s Innovative Genomics Institute (IGI), a separate UC Berkeley/UCSF nonprofit research center co-directed by Doudna and Weissman, whose mission is to use CRISPR to improve public health. IGI was among institutions whose researchers in April published a study detailing a new universally applicable approach for unbiased identification of off-targets in CRISPR-Cas9.
• GSK’s existing collaborations with genetic information partners—notably direct-to-consumer test developer 23andMe, which last year received a $300 million investment from GSK under a collaboration also designed to speed up therapy development by using genetic data to identify new drug targets.

“With the expertise of Jennifer and Jonathan helping to steer the LGR, I am confident the lab will significantly advance our scientific understanding of the relationship between genes and disease to help find better medicines faster,” Barron added.