CRISPR
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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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Mission Bio raises $70 million to help scale its tech for improving the development of targeted cancer therapies
California-based startup Mission Bio has raised a new $70 million Series C funding round, led by Novo Growth and including participation from Soleus Capital and existing investors Mayfield, Cota and Agilent. Mission Bio will use the funding to scale its Tapestri Platform, which uses the company’s work in single-cell multi-omics technology to help optimize clinical trials for targeted, precision cancer therapies.
Mission Bio’s single-cell multi-omics platform is unique in the therapeutic industry. What it allows is the ability to zero in on a single cell, observing both genotype (fully genetic) and phenotype (observable traits influenced by genetics and other factors) impact resulting from use of various therapies during clinical trials. Mission’s Tapestri can detect both DNA and protein changes within the same single cell, which is key in determining effectiveness of targeted therapies because it can help rule out the effect of other factors not under control when analyzing in bulk (i.e. across groups of cells).
Founded in 2012 as a spin-out of research work conducted at UCSF, Mission Bio has raised a total of $120 million to date. The company’s tech has been used by a number of large pharmaceutical and therapeutic companies, including Agios, LabCorp and Onconova Therapeutics, as well as at cancer research centers including UCSF, Stanford and the Memorial Sloan Kettering Cancer Center.
In addition to helping with the optimization of clinical trials for treatments of blood cancers and tumors, Mission’s tech can be used to validate genome editing — a large potential market that could see a lot of growth over the next few years with the rise of CRISPR-based therapeutic applications.
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Pioneering CRISPR researcher Jennifer Doudna is coming to Disrupt
Jennifer Doudna, a woman whose work has triggered the explosion in innovation in the field of synthetic biology and has given researchers around the world a way to program and reprogram the living world, will be speaking at Disrupt in September.
From her positions as the Chancellor’s Chair Professor in the University of California, Berkeley’s Chemistry and Molecular and Cell Biology Departments and a senior investigator at the Gladstone Institutes and professor at the University of California, San Francisco, Doudna has been at the forefront of research into CRISPR gene editing technology.
It was only eight years ago that Doudna and Emmanuelle Charpentier first proposed that CRISPR-Cas9 enzymes (which direct immune responses in microbes) could be used to edit genomes. That discovery would prove to be one of the most significant advancements in the history of the human understanding of biology, and it has the potential to reshape the world.
Doudna describes her own journey into the field of biochemistry beginning back in Hawaii with the discovery of James Watson’s book “The Double Helix” on her father’s bookshelf. From an early age growing up in Hawaii as the daughter of a literature professor, Doudna knew she wanted to pursue a career in science. But it was Watson’s famous book that opened her eyes to the human side of science.
Now her scientific research and startup endeavors have the potential to open humanity’s eyes to the potential benefits of this revolutionary field of science. Because in addition to her research work, Doudna is also a co-founder of a number of companies including: Mammoth Biosciences, Caribou Biosciences, Intellia Therapeutics and Editas Medicine.
These companies are tackling some of the biggest challenges that the world faces. Mammoth is working on a new type of COVID-19 test, Caribou is pursuing novel cancer therapies, and publicly traded Editas is pursuing treatments for ocular, neurodegenerative, and blood diseases as well as cancer therapies.
There’s almost no industry where gene editing hasn’t had some sort of effect. From material science to food science and agriculture to medicine, CRISPR technology is creating opportunities to remake entire industries.
Genetically modified organisms are already making Impossible Foods meat replacements taste meaty; they’re used in Solugen’s bio-based chemicals; and CRISPR edited cells have been proven safe in early trials to treat certain kinds of cancer.
Given the breadth of applications and the questions that the technology’s application raises about how and what limitations researchers should put on the technology, there will be plenty for Doudna to discuss on the Disrupt stage, including but certainly not limited to her recently announced work on making college campuses safer via a fast saliva-based COVID-19 test.
Disrupt is all virtual in 2020 and runs September 14 to September 18, and we have several Digital Pass options to be part of the action or to exhibit virtually, which you can check out here.
Doudna joins an incredible line-up of Disrupt speakers including Sequoia’s Roelof Botha and Atlassian co-founder Mike Cannon-Brookes. We’ll be announcing even more speakers over the coming weeks, so stay tuned.
(Editor’s Note: We’re watching the developing situation around the novel coronavirus very closely and will adapt as we go. You can find out the latest on our event schedule plans here.)
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Mammoth Biosciences aims to be Illumina for the gene editing generation
In 1998, the startup company Illumina launched a revolution in the life sciences industry by developing technology to slash the costs of identifying and mapping genetic material.
Now, a little over 20 years later, Mammoth Biosciences is hoping to do the same thing for gene editing tools.
The company, co-founded by Jennifer Doudna, who did some of the pioneering work to discover the gene editing enzyme known as CRISPR, has just raised $45 million as it looks to bring to market products that can be used not only for disease detection, but are more precise editing tools for genetic material.
Rather than get bogged down in the patent dispute that raged over the provenance and ownership of applications for the original CRISPR enzyme — the Cas9 discovered by Doudna and developed for clinical applications at the Broad Institute — Mammoth has joined a number of startups in identifying new enzymes with a broader array of properties.
“From the very beginning of the company we’ve only worked with novel new enzymes to create these diagnostic products and the new novel diagnostic and editing,” says Trevor Martin, Mammoth Biosciences co-founder and chief executive.
Chiefly, the company is touting its Cas14 enzyme, which the company says opens up new possibilities for programmable biology thanks to its small size, diverse targeting ability and high fidelity — meaning that there are no unforeseen side effects to edits made using the enzyme (something that has arisen with Cas9 applications).
“There’s not one protein that’s going to be the best at everything,” says Martin. “For any particular product that you’re building, at Mammoth, we have the broadest toolbox.”
The Cas14 enzyme can be used to make gene edits in-vivo, meaning in live organisms, instead of ex-vivo, or outside of an organism. The in-vivo use-case could accelerate the time it takes to conduct experiments or develop treatments.
“Twenty years from now, when the umpteenth drug gets approved using Crispr and some nuclease named Cas132013, people are going to look back on this patent battle and think, ‘what a godawful waste of money,’ ” Jacob Sherkow a patent law scholar at New York Law School told Wired back in 2018.
Already, Horizon Discovery, a Cambridge, U.K.-based gene editing technology developer, is using the new tools developed by Mammoth Bioscience to create new CRISPR tools for Chinese Hamster Ovary cell line editing.
That partnership is an example of how Mammoth is thinking about the commercialization of the new Cas14 enzyme line and its role in biological engineering.
“You will need a full toolbox of CRISPR proteins,” says Martin. “That will allow you to interact with biology in the same way that we interact with software and computers. “From first principles, companies will programmatically modify biology to cure a disease or decrease risk for a disease. That’s going to be really kind of a turning point.”
To achieve its vision, Mammoth has managed to nab top talent from the life sciences industry, including Peter Nell, a co-founder of Casebia (a joint venture between Bayer and CRISPR Therapeutics), who came on board as chief business officer, and Ted Tisch, a former executive at Synthego and Bio-Rad, who joined the company as chief operating officer.
The company also nabbed $45 million of funding, including investment firms Mayfield, NFX, Verily (the Alphabet subsidiary) and Brook Byers, which was led by Decheng Capital — bringing the company to more than $70 million in funding.
“There are a dozen or so products that are in clinical development with CRISPR,” says Ursheet Parikh, a partner with Mayfield. “Maybe that number would go up by five or 10 without Mammoth, but it will go up by one or two orders of magnitude with Mammoth.”
To Parikh, Mammoth is the best positioned of the CRISPR development tools, because the company is building a whole platform that customers can license and use to develop products using gene editing.
The thinking, according to Parikh, is as follows, “if this technology can power lots of applications, let’s basically ensure that lots of these applications can come to market and as that happens I get my app store cut.”
“It’s an Illumina-like business,” Parikh says. “Just as anybody who is innovating with genomics needs an Illumina sequencer because they want to be able to do the sequencing… if someone wants to do editing… This gives them the access to do the right sequencing.”
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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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DNA Script picks up $38.5 million to make DNA production faster and simpler
DNA Script has raised $38.5 million in new financing to commercialize a process that it claims is the first big leap forward in manufacturing genetic material.
The revolution in synthetic biology that’s reshaping industries from medicine to agriculture rests on three, equally important pillars.
They include: analytics — the ability to map the genome and understand the function of different genes; synthesis — the ability to manufacture DNA to achieve certain functions; and gene editing — the CRISPR-based technologies that allow for the addition or subtraction of genetic code.
New technologies have already been introduced to transform the analytics and editing of genomes, but little progress has been made over the past 50 years in the ways in which genetic material is manufactured. That’s exactly the problem that DNA Script is trying to solve.
Traditionally, making DNA involved the use of chemical compounds to synthesize (or write) DNA in chains that were limited to around 200 nucleotide bases. Those synthetic pieces of genetic code are then assembled to make a gene.
DNA Script’s technology holds the promise of making longer chains of nucleotides by mirroring the enzymatic process through which DNA is assembled within cells — with fewer errors and no chemical waste material. The enzymatic process can accelerate commercial applications in healthcare, chemical manufacturing and agriculture.
“Any technology that can make that faster is going to be very valuable,” says Christopher Voigt, a synthetic biologist at the Massachusetts Institute of Technology in Cambridge, told the journal Nature.
DNA Script isn’t the only company in the market that’s looking to make the leap forward in enzymatic DNA production. Nuclear, a startup working with Harvard University’s famed geneticist, George Church, and Ansa Bio, a startup affiliated with Jay Keasling’s Berkeley lab at the University of California, are also moving forward with the technology.
But the Paris-based company has achieved some milestones that would make its technology potentially the first to come to market with a commercially viable approach.
At least, that’s what new investors LSP and Bpifrance, through its Large Venture fund, are hoping. They’re joined by previous investors Illumina Ventures, M. Ventures, Sofinnova Partners, Kurma Partners and Idinvest Partners in backing the company’s latest funding.
The company said the money would be used to accelerate the development of its first products and establish a presence in the United States.
“As we announced earlier this year at the AGBT General Meeting, DNA Script was the first company to enzymatically synthesize a 200mer oligo de novo with an average coupling efficiency that rivals the best organic chemical processes in use today,” said Thomas Ybert, chief executive and co-founder of DNA Script. “Our technology is now reliable enough for its first commercial applications, which we believe will deliver the promise of same-day results to researchers everywhere, with DNA synthesis that can be completed in just a few hours.”
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Mushroom leather, tiny Zika detectors and lab-made breast milk debut at IndieBio’s third demo day
The accelerator’s demo day has grown so big we’re now live streaming it on TechCrunch. But it was just a couple years ago that the only pure biotech accelerator launched out of SOS Ventures. Many accelerators and venture firms have started to take a keen interest in the space since then, but Indiebio is still the one many look to in the industry for weird and interesting ideas like… Read More
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