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CAR-T cell therapy, produced from the blood of healthy donors, that targets blood-borne cancers. For its therapeutic work, Caribou is using “a number of different gene editing technologies” that are not licensed to Intellia, Haurwitz said.
Doudna is one of Caribou’s two scientific advisors; the other is Martin Jinek, a former member of Doudna’s lab and co-author of the seminal 2012 paper, with Doudna and French microbiologist Emmanuelle Charpentier, that describes the work they did to turn CRISPR-Cas9 into a gene-editing tool.
Off-the-shelf, or allogeneic, CAR-T refers to cell-based therapies that use blood-based T cells from healthy donors. Genetic alterations turn the T cells into revved-up cancer killers that, when given to a cancer patient, attack the patient’s tumor but don’t trigger a dangerous immune rejection. That’s the goal, at least. Two CAR-T medicines derived from a patient’s own cells have been approved in the U.S., but the first off-the-shelf CAR-Ts have only begun human testing recently. Caribou will face much larger competitors, in particular the well-funded Allogene (NASDAQ: ALLO).
For years, Caribou has signaled its intent to develop its own cell therapies, hiring biopharma veterans for its executive ranks. Announcing the 2017 arrival of current chief scientific officer Steven Kanner—previously of Bristol-Myers Squibb, Arrowhead Pharmaceuticals, and other firms—a press release included this quote from Haurwitz: “He will also be an important leader in generating opportunities for our rapidly emerging pipeline of off-the-shelf CAR-T candidates and microbiome-based therapies.”
Beyond what UC Berkeley transferred from Doudna’s lab, Caribou has amassed its own patent portfolio, both from its internal work and from other industrial and academic entities such as Dupont.
Caribou now has 55 employees and has raised more than $40 million in private investment. Despite the unprecedented IPO biotech boom in the past five years, the firm has stayed private.
Asked about that last week, Doudna said, “Maybe it would’ve been better to be bigger and go public.”
Then again, the Intellia stake has given Caribou resources it wouldn’t otherwise have, Doudna said. The firm’s evolution has required a team that’s “good at cell-based therapy, or any clinical application,” that can put together labs for animal studies, and think about manufacturing and pricing—skills required for a biotech pushing its own products into clinical trials and eventually toward the market. Without public shareholder pressure, that team has also been free to pursue more cutting-edge projects, such as editing microbial DNA. “They can take that risk,” she said.