In New Gene-Editing World, Doudna Applies Both Brake and Gas Pedal

working on the latter? Or that by making this distinction in your letter, you’re sending a mixed signal?

JD: No. I think we clearly stated that research in these systems should proceed in order to inform future discussion and decision-making regarding possible clinical applications.

X: Will you be doing basic research in germline editing at the IGI?

JD: None is planned at the present time.

X: How did IGI come about?

JD: The IGI started with a vision of creating a venue for connecting basic and clinical scientists in the Bay Area to develop and apply powerful new genome engineering technology to address human disease. We also wanted to foster entrepreneurship and partnerships between companies and academic labs.

X: Is IGI an umbrella for work ongoing at Berkeley and UCSF, or will IGI carve out its own definable research?

JD: IGI is carving out its own areas of research, focused initially on platform technology development and applications to understanding and treating diseases of the immune system.

X: How do you plan to staff up beyond the folks currently listed on the Website?

JD: IGI has $10M from the Li Ka Shing Foundation and $1M each from UC Berkeley and UCSF. We also have funds from ongoing deals with AstraZeneca and Agilent Technologies. And we are actively seeking additional donors and corporate partnerships to fund specific projects.

X: Will IGI spin out work via the Berkeley and UCSF tech transfer offices? Will it have its own commercialization staff?

JD: IP generated within the IGI will be filed through the UC Regents, and in some cases may be jointly owned with corporate partners. As the IGI grows, it may eventually make sense to employ in-house commercialization staff.

X: Do you have any rules for working with industry?

JD: We seek partners who want to engage with Bay Area academic labs and/or with the IGI research team. Terms of such agreements can include joint ownership of newly-developed IP, as well as opportunities to work closely on projects of mutual interest. We invite companies to visit us and meet our scientists.

X: The website gives a few examples of research IGI will pursue: Why have you chosen those projects?

JD: These projects, on ubiquitin signaling pathways and protein kinases, are ideal for honing the capabilities of the CRISPR-Cas9 technology for genome manipulation. Jacob Corn, the scientific director of the IGI, has experience in both of these areas, and we also anticipate interest from corporate partners that will help expand the IGI’s research program.

X: IGI will also work on a “nextome” project—looking for disease-causing genes in the vast portion of DNA (sometimes known as “junk DNA”) that don’t seem to carry code that turns into proteins—which builds upon sequencing work already in progress at UCSF. How will you layer CRISPR-Cas9 technology on top of that work?

JD: The project builds on research underway in the lab of Jennifer Puck at UCSF. We plan to use CRISPR-Cas9 to determine the underlying genetic basis for immune disorders in pediatric patients, and in the longer term, to develop effective strategies for cell-based treatment of these disorders using a personalized approach.

X: Could you give an example of a pediatric disease with known or suspected genetic drivers in the nextome?

JD: In general, we are focused on severe combined immune-deficiency (SCID)-type disorders, for which about 70 percent have a genetic basis that has yet to be determined.

 

Author: Alex Lash

I've spent nearly all my working life as a journalist. I covered the rise and fall of the dot-com era in the second half of the 1990s, then switched to life sciences in the new millennium. I've written about the strategy, financing and scientific breakthroughs of biotech for The Deal, Elsevier's Start-Up, In Vivo and The Pink Sheet, and Xconomy.