Tag: Editas Medicine

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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.

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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Up to $818 million deal between J&J and Locus Biosciences points to a new path for CRISPR therapies

The up to $818 million deal between Locus Biosciences and Janssen Pharmaceuticals (a division of Johnson & Johnson) that was announced yesterday points toward a new path for CRISPR gene editing technologies and (potentially) the whole field of microbiome-targeted therapies.

Based in Research Triangle Park, N.C., Locus is commercializing research initially developed by scientists at North Carolina State University that focused on Cas3 proteins, which devour DNA Pac-Man-style, rather than edit it like the more well-known Cas9-based CRISPR technologies being used by companies like Caribou Biosciences, Editas Medicine, Synthego, Intellia Therapeutics, CRISPR Therapeutics and Beam Therapeutics.

While the Cas9 CRISPR technologies can edit targeted DNA — either deleting specific genetic material or replacing it with different genetic code — Cas3 simply removes DNA strains. “Its purpose is the destruction of invading DNA,” says Locus chief executive, Paul Garofolo.

The exclusive deal between Janssen Pharmaceuticals and Locus gives Janssen the exclusive license to develop, manufacture and commercialize CRISPR-Cas3-enhanced products targeting bacterial pathogens for the potential treatment of respiratory and other organ infections.

Under the terms of the deal, Locus is getting $20 million in upfront payments and could receive up to $798 million in potential future development and commercial milestone payments and any royalties on potential product sales.

A former executive at Valiant Pharmaceuticals and Paytheon, Garofolo was first introduced to the technology that would form the core of Locus as an executive in residence at North Carolina State University. It was there that he met Dr. Chase Beisel and Rodolphe Barrangou, whose research into Cas3 proteins would eventually be productized by Locus.

The company spun out of NC State in 2015 and raised its first cash from the North Carolina Biotech Center a year later.

Locus is already commercializing a version of its technology with bacteriophages designed to target e coli bacteria to treat urinary tract infections. The company is on target to begin its first clinical trials in the third quarter of the year.

The focus on bacterial infection and removing harmful bacteria while ensuring that the rest of a patient’s microbiome is intact is a huge step forward for treating diseases that scientists believe could be linked to bacterial health in a body, according to Garofolo.

“Most microbiome companies are about adding probiotics to your body,” says Garofolo, representing a thesis that introducing “good” bacteria to the body can offset any harmful pathogens that have infected it.

“Things you’re exposed to are creating the groundwork for an infection or disease, or exacerbating an existing disease,” says Garofolo. And while he believes that the microbiome is the next big field for scientific discovery, the approach of adding probiotics to a system seems less targeted and effective to him.

Already, Garofolo has managed to convince investors of his approach. In addition to the initial outside investment from the North Carolina Biotech Center, Locus has attracted $25 million in financing from investors, including Artis Ventures and the venture capital arm of the Chinese internet giant, Tencent.

Meanwhile, investors have spent millions backing alternative approaches to improving human health through the manipulation of the microbiome.

Companies like Second Genome, Viome and Ubiome are all using approaches that identify bacteria in the human body and try to regulate the production of that bacteria through diet and probiotic pills. It’s an approach that allows these companies to skirt the more stringent requirements the Food and Drug Administration has put in place for drugs.

That doesn’t mean that extensive amounts of research haven’t gone into the development of these probiotics. Seed, a Los Angeles-based startup that launched last year, has recruited as its chief scientist George Reid, the leading scientist on microbial health and the microbiome.

Founded by Raja Dhir, a graduate from the University of Southern California and a leading researcher on microbiotics in his own right, and Ara Katz, the former chief marketing officer of BeachMint and an MIT Media Lab fellow, Seed focuses on developing probiotic treatments using well-established research.

“Foundational to our approach is that it’s not which microbes are present in your gut… It’s based on looking at what specific microbes can do to a healthy individual to improve that status of health independent of what is already present,” Dhir said in an interview around the company’s launch last June. “It’s a little bit less exciting from a tech perspective, but it’s hardcore grounded in basic science… The question is, does this have changes and effects in validated bio-makers in a controlled and placebo setting?”

Dhir said that a basic understanding of how different bacteria can influence health is necessary before getting into the benefits of personalization.

“These things can dance between drugs and nutrition,” Dhir said. “Probacteria are an additional lever that people should pull… like diet and exercise and cessation of smoking… In every correspondence we always have been and need to be clear that this should never be seen as a replacement of therapies.”

By contrast, the tools that Locus is developing are very much therapies with potentially far-reaching implications for illnesses, from irritable bowel syndrome to gastrointestinal cancers and even neurological disorders.

“The science [around the microbiome] is early, but it is very well-known that a potentially deadly pathogen should be removed from your body,” Garofolo said.