Young and poised, Rachel Haurwitz showed up for an interview
at the IBC Boston Gene Editing meeting right on schedule.
Every inch a corporate executive, deftly fielding questions, graciously skirting controversial issues, she clearly laid out the corporate development and strategic planning for Caribou Biosciences. It’s only when she got into the nitty gritting of biology that the mask slipped a bit.
Haurwitz is a PhD. a biochemist with a structural biology background. She studied biology and CRISPR in Jennifer Doudna’s lab at UC Berkeley in 2007 in the very earliest days of CRISPR development. She’s also president and CEO of Caribou Biosciences, a company she co-founded along with Doudna, to support the commercial applications of CRISPR technology. That makes her a partner in two stratospheric levels of competitive, high-impact technology: Drug development with Novartis and biology with Doudna.
“So, we have a couple of different CRISPR based technologies at Caribou. The main focus today of course is the Cas9 genome editing platform,” she said and right here she slipped back into her laboratory bench persona as she warmed to her subject. “…and other CRISPR based technologies. One is acidity tagging and purifying RNAs and RNA protein complexes, a capability we use as a stand alone biology tool and something we use in conjunction with the CAS9 platform.”
The way Caribou works, says Haurwitz, is to focus, “as a platform technology company” in four major areas, therapeutics, research, agriculture and industrial biology. Access to product development and commercial markets is gained through strategic collaboration. The Novartis collaboration is designed to develop CRISPR based technology for drug target evaluation and validation screening.”
One of those collaborations resulted from a Caribou spinoff, the formation of Intellia last year with participation by Novartis and venture firm, Atlas. Again the issue is focus. “”From our perspective the skills it takes for broad development across plants, humans, and industrial microbes of very different from biomedical research for clinical application. We wanted to have our cake and eat it too
“Intellia is laser focused on gene and cell therapy, product development and roduct advance into the clinic while Caribou maintains the responsibility of improving the platform. That also helps Intellia as we develop new versions of the technology, they will have access to it”
The timeline here is notable. Rachel Haurwitz came to the Doudna lab when in 2007, when she was 21 years old. Her desk was positioned right next to that of Martin Jinek, a Czech post-doc who worked with a Polish researcher in Emmanuelle Charpentier’s Austrian lab on CRISPR issue. Jinek, who was born close to the Polish border would slip into Polish during his intercontinental conversations. It was during one of those exchanges in Polish that the implications of the Cas9 enzyme as a nuclease associated with CRISPR emerged. By 2012, Doudna and Charpentier published their findings and the CRISPR revolution was launched.
Within a year Rachel Haurwitz was tapped by Jennifer Doudna to head up Caribou and help realize its practical potentials. The following year, Intellia was spun off and millions of dollars were invested in both entities while Caribou built its capabilities. “Now we are 20 people with a heavy emphasis on R&D. There’s a little more hiring coming up but that will be project and product specific as we ramp up capacities.”
If that last little bit of CEO-speak seemed to reveal the real Rachel Haurwitz (“I haven’t touched a pipette in years.”) that impresson would be wiped out the following morning when she delivered her presentation on CRISPR designs for Cas9-mediated genome engineering.
She talked about engineering particular elements of the guide RNA to increase targeting specificity and then proceeded to break down the CRISPR into six distinct functional modules, upper stem, lower stem, repeat, short hairpin… etc Haurwitz was describing means of increasing the cleavage efficiency of the CRISPR molecule by understanding what was essential in its operation and what wasn’t. When she got to increased transfection and efficiency by editing with an engineered protein versus a plasmid all vestiges of the cool CEO describing strategic collaborations were gone.
This was a scientist on the frontiers of biology delivering her findings to a darkened roomful of scientists.
That afternoon, along with an all star panel of high level researchers and chief scientific officers, she participated in a panel discussion: Genome editing nuclease specificity: Defining off-target effects using genome editing vong for therapeutic applications. You had to have been there.
And she’s still in her ‘twenties.
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