New CRISPR Lab to Accelerate Drug Discovery, Advance Genomic Research

'Laboratory for Genomic Research' Unites CRISPR Pioneers with Industry Expertise to Unravel Mysteries of Human Genome

Drug Discovery
Jennifer Doudna, PhD, and Jonathan Weissman, PhD. Photo by Barbara Ries

GlaxoSmithKline plc (GSK) has launched a five-year, $67 million collaboration with the San Francisco and Berkeley campuses of the University of California to build a state-of-the-art laboratory that will use CRISPR technologies to explore how genes cause disease and to rapidly accelerate the discovery of new medicines.

Known as the Laboratory for Genomic Research (LGR), the partnership will be headed by Dr. Hal Barron, chief scientific officer and president, R&D, at GSK; UCSF’s Jonathan Weissman, PhD, professor of cellular and molecular pharmacology, who has pioneered new applications of CRISPR for biological research; and CRISPR co-inventor Jennifer Doudna, PhD, professor of biochemistry, biophysics and structural biology at UC Berkeley.

The new lab will be housed on Illinois Street adjacent to UCSF’s Mission Bay campus. Once fully established, it will provide facilities for 24 University employees funded by GSK as well as up to 14 GSK employees, with a focus on immunology, neuroscience, and oncology. GSK’s machine learning group will build computational pipelines to analyze the extensive data the LGR will produce.

“Over the last seven years, CRISPR has transformed academic research, but until the LGR, we haven’t had a focused effort to catalyze the kind of research we know will lead to new innovation using this CRISPR tool,” said Doudna. “The LGR is about building that space where creative science is partnered with the development of robust technology that will help develop tomorrow’s drugs. I think we’re going to be able to do science that none of us can even imagine today.”

With the recent explosion of information from human genetics, scientists need powerful tools to understand why small changes in a person’s genetic makeup can increase the risk of diseases, an area of science called functional genomics. The most powerful tool in functional genomics, CRISPR, allows this to be done at a scale once thought impossible. Through this research, scientists can discover and develop novel therapies that have a higher likelihood of becoming medicines.

“Technology is key to our innovation strategy at GSK, and CRISPR is one of the most important technologies of our time,” said Barron. “With the expertise of Jennifer and Jonathan helping to steer the LGR, the mission of the lab is to advance our scientific understanding of the relationship between genes and disease to help find better medicines faster.”

The LGR represents a novel hybrid model that brings together industrial and academic researchers under a single roof working on projects both together and independently.  The outputs of those research projects will be focused on technologies, new drug targets and biological mechanisms that will foster both academic and industrial advances.

The new laboratory will also be a resource for investigators at both UC campuses, who can access and use its technology to answer their own biomedical or other biological questions, and to develop new tools that explore how genes work.

“The LGR screening center will be available to labs at UCSF and Berkeley,” Weissman said, “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.”

The LGR will receive up to $67 million in funding over a five-year period, which will include facilities for 24 full-time university employees funded by GSK, plus up to 14 full-time GSK employees. With a focus on immunology, oncology and neuroscience, the laboratory will be adjacent to the UCSF Mission Bay campus in San Francisco.

GSK’s artificial intelligence and machine learning group will also be involved in building the necessary computational pipelines to analyze data. The LGR aims to automate and scale up existing CRISPR approaches so that this work can be done much more rapidly. Ultimately the goal is to deepen our understanding of genetics and discover new targets, and to create next-generation technologies that will become future standard practice for pharma industry.

In keeping with UC’s public mission, the tools that are developed in the lab will be described in published papers, subject to intellectual property provisions, and will be available for use by other academic and non-profit labs.

“One of our key goals is to advance the field overall and make these tools as broadly available as possible,” Weissman said.

The collaboration will be governed by a Joint Steering Committee with equitable University and GSK representation, and additional joint sub-committees covering patents, scientific and project management. Doudna and Weissman will be members of the Joint Steering Committee, together with GSK’s new head of functional genomics, Christopher Miller, PhD.

The project builds on the work of the Innovative Genomics Institute, a separate UC Berkeley/UCSF nonprofit research center co-directed by Doudna and Weissman, which aims to use CRISPR to improve public health.

The collaboration will also build on GSK’s existing collaborations with companies such as 23andMe, which delivers genetic information at scale. By applying artificial intelligence and machine learning to identify correlations between genetic variants and disease, researchers can select for clinical trials those patients who are most likely to benefit, thereby speeding up the drug development process.