Genetic Engineering & Biotechnology News

AUG 2018

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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14 | AUGUST 2018 | Genetic Engineering & Biotechnology News | GENengnews.com Although CRISPR pioneers are few—names such as Jen- nifer Doudna, Feng Zhang, and George Church come to mind—they accomplish so much that they seem to be in several places at once, directing expansive research agendas here, and participating in diverse commercial ventures there. Four to five years ago, CRISPR pioneers helped launch the first group of CRISPR ventures, most notably Intellia Therapeutics, CRISPR Therapeutics, and Editas Medicine. As these names indicate, the first CRISPR ventures started by emphasizing therapeutic applications. In contrast, new- er CRISPR ventures are striking out in multiple directions. Besides exploring therapeutics, the new startups, many of which are also associated with CRISPR pioneers, are look- ing into diagnostics, agriculture, and other application areas. For some time now, the CRISPR pioneers have been on each other's heels. Even before joining the first group of CRIS- PR ventures, these scientists were publishing the breakthrough papers that gave CRISPR a new meaning. Initially, CRISPR (clustered regularly interspaced short palindromic repeats) emerged as a genomic pattern emblematic of bacterial im- mune systems. Then, thanks to the CRISPR pioneers, CRISPR came to stand for bacterial immune system elements that, upon reengineering, may serve as powerful gene editing tools. For example, several CRISPR-associated (Cas) nucleases have emerged, each endowed with distinctive capabilities. Currently, the CRISPR pioneers are starting new ascents of Mount Commercialization. Rising from their base camps, the CRISPR pioneers may soon be on each other's heels again—or not, if they follow different paths. There is, as this article illustrates, a lot of ground to cover. Mammoth Biosciences One new company researching and developing CRISPR ap- plications in the therapeutic space is Mammoth Biosciences, headquartered in San Francisco, CA. Notable researcher and CRISPR pioneer, Jennifer Doudna, Ph.D., serves as both the co- founder and chair of the company's scientific advisory board. According to Mammoth's CEO and co-founder, Trevor Martin, Ph.D., Mammoth was created to democratize diagnos- tics that rely on genetic information. "Right now, there are a lot of trends in medicine, especially in personalized medicine and tailored therapies," says Dr. Martin. "In general, we think the industry is shifting toward early and improved diagnostics." The researchers at Mammoth are searching for novel func- tions of CRISPR that go beyond identifying viruses, bacteria, and disease-related genetic mutations. For these researchers, the ultimate goal is the creation of an in-home genetic test- ing device that can provide point-of-care disease detection. These researchers assert that the company's device, which is still in its prototype phase, will retain design elements that are meant to support economy and user friendliness. "At Mammoth, we have the world's most affordable and easy-to-use programmable system for performing diagnos- tics at the molecular level," Dr. Martin insists. Although Mammoth's CRISPR-based platform has yet to enter routine clinical practice, the company anticipates that its technology will revolutionize the way diagnostic tests are performed. Instead of going to the doctor's office, patients can use Mam- moth's technology to identify health issues at home, thereby improving healthcare access and reducing the time spent trav- eling to and from testing facilities. Patients using the technol- ogy start by placing a fluid sample on a paper strip. Then the patient uses a smartphone to take a picture of the paper strip to capture any color change that may occur. Next, the patient uploads the picture to Mammoth's "companion app," which interprets the image and generates a test result. According to Mammoth, the wait for a test result lasts just 30 minutes. "One of the reasons we're really excited about this plat- form is its programmability," Dr. Martin declares. "It's al- ways the same protein we're using in every test, and with that we're actually switching out the guide RNA." An additional feature of the Mammoth platform, according to Dr. Martin, is its ability to detect the presence of a pathogen as well as identify the detected pathogen's specific strain. Beam Therapeutics Another CRISPR pioneer, Feng Zhang, Ph.D., of the Broad Institute of MIT, is credited with co-founding Beam Thera- peutics, a company whose primary focus is on base editing for altering sequences of DNA. This new precision medicine com- pany recently secured $87 million in Series A funding from F-Prime Capital Partners and ARCH Venture Partners. Using these funds, Beam hopes to focus on base-editing platforms. Such platforms enable the direct, irreversible conversion of one base pair to another at a target genomic locus. Currently, Beam has licensing agreements for two base- editing platforms developed by Harvard researcher David Liu, Ph.D. One platform is a C base editor, which delivers programmable C-to-T or G-to-A edits in DNA. The other platform is an A base editor, which delivers programmable A-to-G or T-to-C edits. Additionally, the company licenses an RNA editor plat- form developed in Dr. Zhang's laboratory. Beam did not comment on its plans for this platform when it was con- tacted by GEN, but since it emerged from stealth mode, the company indicated that it will develop a pipeline of precision genetic therapies that repair disease-causing point mutations. Pairwise Plants In early 2018, Monsanto (now a part of Bayer) invested $125 million in Pairwise Plants, a startup cofounded by Dr. Zhang, Dr. Liu, and fellow CRISPR stalwart Keith Joung, Ph.D. The company was built around the idea of CRISPR technology, specifically base editing technology, to deliver ag- ricultural gene editing services. Monsanto's gene editing strategy lead, Scott Knight, Ph.D., tells GEN that the Pairwise-Monsanto venture was intended to enable the biotech giant to accelerate the use of gene editing in the top five agricultural plants: corn, soy, cotton, canola, and wheat. Pairwise-Monsanto's plans included the development of plants possessing the ability to resist disease and tolerate drought or flood, as well as the ability to produce flavorful food. CRISPR is exciting because it gives developers the ability to go into the genome to create new variation. "But to use the tool effectively," Dr. Knight advises, "you have to do the work be- OMICS The ultimate goal for a number of CRISPR researchers is the creation of an in-home genetic testing device that can provide point-of-care disease detection. vchal/Getty Images New CRISPR Startups Get a Leg Up Brandon May CRISPR startups, like all high-tech startups, climb Mount Commercialization faster if they get an extra boost, which may come from a unique technology, a compelling application, or a hoard of venture capital. Some CRISPR startups, however, benefit from another sort of boost—the presence and guiding intelligence of a true CRISPR pioneer.

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