GSK and CRISPR Pioneer Jennifer Doudna Launch Genomics Research Lab to Accelerate Drug Discovery

Arterial New Medicine (WeChat ID: biobeat1) learned from foreign media that on June 17, the world-renowned biotechnology company GlaxoSmithKline (GSK) announced the signing of a five-year collaborative agreement with the University of California (UC). The two parties will jointly establish an advanced laboratory to explore the intrinsic pathological mechanisms by which gene mutations cause diseases, and are committed to developing new CRISPR gene-editing technologies to accelerate the research and development of novel therapies.

It is reported that Dr. Jennifer Doudna of the University of California, Berkeley, and Dr. Jonathan Weissman of the University of California, San Francisco, will join the project, working alongside Dr. Hal Barron, Vice President of R&D at GSK, to launch the Laboratory for Genomics Research (LGR).

Dr. Jennifer Doudna is the co-inventor of CRISPR gene-editing technology and is hailed as a leading figure in the “CRISPR revolution.” In 2012, she and Professor Emmanuelle Charpentier first proposed that CRISPR/Cas9 could be used for programmable editing of genomes, marking one of the most significant discoveries in the history of biology. Dr. Doudna has received numerous awards for her achievements, including the Canada Gairdner International Award in 2016, which she shared with Chinese scientist Feng Zhang.

Dr. Jonathan Weissman is a pioneer in CRISPR screening technology and holds a Ph.D. in Physics from Harvard University. His research primarily focuses on ensuring that proteins fold into their correct conformations and investigating the roles of misfolded proteins within the body.

Dr. Jonathan Weissman’s laboratory is currently developing a modified version of CRISPR technology known as CRISPRa, and last year demonstrated its ability to reverse genetic forms of severe obesity in two different mouse models. CRISPRa employs the CRISPR guide system to target specific gene sequences; however, instead of using Cas enzymes as molecular scissors to cut or replace DNA sequences, this technique amplifies existing gene activity to increase protein production.

Under this collaboration agreement, GSK will provide $67 million in funding over five years to support the construction of the LGR, and will supply relevant R&D equipment for 24 full-time university staff members and 14 full-time GSK employees.

GSK stated that it would deploy its artificial intelligence and machine learning teams to help LGR develop the computational tools necessary for generating data, with the aim of enabling LGR to automate CRISPR gene editing. This would facilitate large-scale genetic analyses to identify associations between genetic variants and diseases. Meanwhile, LGR researchers would be responsible for selecting patients most likely to benefit for clinical trials, thereby accelerating the development of new therapies.

According to the latest statements from GSK and UC, the LGR will bring together researchers from industry and academia to collaborate on a shared research project. This project will focus on the development of technologies and new drugs, promoting mutual advancement between academia and industry.

Dr. Jennifer Doudna stated, “The founding of LGR represents a powerful combination of creative science and robust development technologies, which will help us reach new scientific heights and develop novel therapeutics.”

“Our goal is to comprehensively advance the field of gene editing and provide widely accessible related tools wherever possible,” added Dr. Jonathan Weissman. “LGR will provide relevant support to laboratories at the University of California, San Francisco (UCSF) and the University of California, Berkeley, enabling research that cannot be independently conducted in their own labs to be accomplished with LGR’s technical support.”

Dr. Hal Barron stated, “With the strong support of Dr. Jennifer Doudna and Dr. Jonathan Weissman, I am confident that LGR will significantly advance our scientific understanding of the relationship between genes and disease, thereby accelerating the development of better therapeutics.”

Currently, LGR’s work will be built upon two foundations: first, the Innovative Genomics Institute (IGI) at UC, which has developed a method capable of unbiasedly identifying off-target effects in CRISPR-Cas9 systems; and second, GSK’s partnership with 23andMe. Last year, GSK invested $300 million in 23andMe, a consumer-grade genetic sequencing developer, aiming to accelerate the development of related therapies by leveraging genetic data to identify new drug targets.

Furthermore, representatives appointed by GSK and UC will join the Joint Steering Committee to oversee this collaboration, while other joint teams will be responsible for patents, scientific management, and project management. Doudna, Weissman, and Dr. Chris Miller will join the Joint Steering Committee. GSK and UC further stated that relevant technical tools developed in the future under the LGR initiative will be made available to other academic and non-profit laboratories, subject to intellectual property protection.

It is reported that the laboratory will be located on Illinois Street in San Francisco, near the University of California, San Francisco’s Mission Bay campus.

(Compiled by Wang Chan)