genetic engineering
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New clinical trial data from Locus Biosciences shows promise in CRISPR-Cas3 technology
Antibiotic resistance is one of the biggest potential threats to global health today. But Locus Biosciences is hoping that their crPhage technology might provide a new solution.
Based in North Carolina’s Research Triangle, the startup recently announced promising phase 1b clinical trial results for their use of CRISPR-Cas3-enhanced bacteriophages as a treatment for urinary tract infections caused by escherichia coli. Led in part by former Patheon executive and current Locus CEO Paul Garofolo, the startup launched in 2015 with the goal of using a less popular application of CRISPR technology to address growing antimicrobial resistance.
CRISPR-Cas3 technology has notably different mechanisms from its more well-known CRISPR-Cas9 counterpart. Where the Cas9 enzyme has the ability to cleanly cut through a piece of DNA like a pair of scissors, Garofolo describes Cas3 more like a Pac-Man, shredding the DNA as it moves along a strand.
“You wouldn’t be able to use it for most of the editing platforms people were after,” he said, noting that meant there wouldn’t be as much competition around Cas3. “So I knew it would be protected for some time, and that we could keep it quiet.”
Garofolo and his team wanted to use CRISPR-Cas3 not to edit harmful bacteria found in the body, but to destroy it. To do this, they took the DNA-shredding mechanism of Cas3 and used it to enhance bacteriophages — viruses that can attack and kill different species of bacteria. Together, co-founder and Chief Scientific Officer Dave Ousterout — who has a PhD in biomedical engineering from Duke — thinks this technology offers an extremely direct and targeted way of killing bacteria.
“We armed the phages with this Cas3 system that attacks E. coli, and that sort of dual mechanism of action is what comes together, essentially, as a really potent way to remove just E. coli,” he said in an interview.
That specificity is something that antibiotics lack. Rather than targeting only harmful bacteria in the body, antibiotics typically wipe out all bacteria they come across. “Every time we take antibiotics, we’re not thinking about all the other parts of us that are impacted by the bacteria that do good things,” said Garofolo. But the precision of Locus Biosciences’ crPhage technology means that only the targeted bacteria would be wiped out, leaving those necessary to the body’s normal function intact.
Beyond offering this more specific approach to treatment of pathogens, or any bacteria-based disease, Garofolo and his team also suspect that their approach will also be extremely safe. Though deadly to bacteria, bacteriophages are typically harmless to humans. The safety of CRISPR in humans is well-established, too.
“That’s our secret sauce,” said Garofolo. “We can build drugs that are more powerful than the antibiotics they’re trying to replace, and they use phage, which is probably one of the world’s safest ways to deliver something into the human body.”
While this new technology could certainly help treat pathogens and infectious diseases, Garofolo hopes that indications in immunology, oncology and neurology might benefit from it too. “We’re starting to figure out that some bacteria might promote cancer, or inflammation in your gut,” he said. If researchers can identify the bacteria at the root cause of those conditions, Garofolo and Ousterout think the crPhage technology might prove to be an effective treatment.
“If we’re right about that, it’s not just about infections or antimicrobial resistance, but helping people overcome cancer or delay the onset of dementia,” Garofolo said. “It’s changing the way we think about how bacteria really help us live.”
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$125 million for Inscripta may usher in the next wave of genetic engineering
In these waning days of the second decade of the twenty-first century, technologists and investors are beginning to lay the foundations for new, truly transformational technologies that have the potential to reshape entire industries and rewrite the rules of human understanding.
It may sound lofty, but new achievements from businesses and research institutions in areas like machine learning, quantum computing and genetic engineering mean that the futures imagined in science fiction are simply becoming science.
And among the technologies that could potentially have the biggest effect on the way we live, nothing looms larger than genetic engineering.
Investors and entrepreneurs are deploying hundreds of millions of dollars to create the tools that researchers, scientists and industry will use to re-engineer the building blocks of life to perform different functions in agriculture, manufacturing and medicine.
One of these companies, 10X Genomics, which gives users hardware and software to determine the functionality of different genetic code, has already proven how lucrative this early market can be. The company, which had its initial public offering earlier this year, is now worth $6 billion.
Another, the still-private company Inscripta, is helmed by a former 10X Genomics executive. The Boulder, Colo.-based startup is commercializing a machine that can let researchers design and manufacture small quantities of new organisms. If 10X Genomics is giving scientists and businesses a better way to read and understand the genome, then Inscripta is giving those same users a new way to write their own genetic code and make their own organisms.
It’s a technology that investors are falling over themselves to finance. The company, which closed on $105 million in financing earlier in the year (through several tranches, which began in late 2018), has just raised another $125 million on the heels of launching its first commercial product. Investors in the round include new and previous investors like Paladin Capital Group, JS Capital Management, Oak HC/FT and Venrock.
“Biology has unlimited potential to positively change this world,” says Kevin Ness, the chief executive of Inscripta . “It’s one of the most important new technology forces that will be a major player in the global economy.”
Ness sees Inscripta as breaking down one of the biggest barriers to the commercialization of genetic engineering, which is access to the technology.
While genome centers and biology foundries can manufacture massive quantities of new biological material for industrial uses, it’s too costly and centralized for most researchers. “We can put the biofoundry capabilities into a box that can be pushed to a global researcher,” says Ness.
Earlier this year, the company announced that it was taking orders for its first bio-manufacturing product; the new capital is designed to pay for expanding its manufacturing capabilities.
That wasn’t the only barrier that Inscripta felt that it needed to break down. The company also developed a proprietary biochemistry for gene editing, hoping to avoid having to pay fees to one of the two laboratories that were engaged in a pitched legal battle over who owned the CRISPR technology (the Broad Institute and the University of California both had claims to the technology).
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Frontier technologies are moving closer to the center of venture investment
As the technologies that were once considered science fiction become the purview of science, the venture capital firms that were once investing at the industry’s fringes are now finding themselves at the heart of the technology industry.
Investing in the commercialization of technologies like genetic engineering, quantum computing, digital avatars, augmented reality, new human-computer interfaces, machine learning, autonomous vehicles, robots, and space travel that were once considered “frontier” investments are now front-and-center priorities for many venture capital firms and the limited partners that back them.
Earlier this month, Lux Capital raised $1.1 billion across two funds that invest in just these kinds of companies. “[Limited partners] are now more interested in frontier tech than ever before,” said Bilal Zuberi, a partner with the firm.
Lux Capital just closed on a whopping $1 billion in capital, doubling the amount of money it manages
He sees a few factors encouraging limited partners (the investors who provide financing for venture capital funds) to invest in the firms that are financing companies developing technologies that were once considered outside of the mainstream.
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