Locus raises $19M to take CRISPR-based antimicrobial ‘Pac-Man’ into clinic

CEO Paul Garofolo says the company's first drug in trials could target C. diff.

North Carolina-based biotech Locus Biosciences is moving ahead with a first clinical trial for its CRISPR-based antimicrobial drugs—armed with $19 million in series A funding.

Plenty of companies are looking at using the gene-editing technology CRISPR-Cas9 to treat human diseases, but Locus is the only company in the world using a different pathway—CRISPR-Cas3—to tackle the pressing problem of multidrug-resistant bacteria, according to CEO Paul Garofolo.

“Selecting the right tool for the job at hand is critical for success,” he tells us. “Both Cas3 and Cas9 are very capable solutions in their own rights, but Cas3 causes irreversible DNA damage—think Pac-Man chewing up the target bacteria’s DNA—resulting in cell death. As such, it is the overwhelming correct choice for an antibacterial product.”

The first-round fundraising—led by Artis Ventures with backup from Tencent Holdings, Abstract Ventures and the North Carolina Biotechnology Center, among others—will fund the two-year-old company through an IND filing and first clinical trial.

Right now, that first trial looks like it will involve Locus’ lead product for recurrent Clostridium difficile infections, but Garofolo says the biotech is “in discussions with several strategic partners regarding C. diff and other assets, so it is possible that priorities could shift based on those discussions.”

Along with C. diff, which is a massive public health problem—the CDC estimates there were 500,000 infections and 29,000 associated deaths in the U.S. in 2011—Locus also has programs looking at CRISPR drugs for infections caused by Pseudomonas aeruginosa and Enterobacteriaceae.

“Locus’ CRISPR-Cas3 technology can be used for the potent removal of specific pathogens, including multidrug-resistant bacteria, while leaving the rest of the microbiome intact,” says Garofolo.

“This highly targeted approach reduces the selective pressure that favors emergence of resistance to traditional antibiotics. Because we target multiple sites within the bacterial genome, it will be much more difficult for the target bacteria to evolve resistance than with a traditional antibiotic.”

The CEO wants to push down hard on the gas to get the CRISPR platform into the clinic and says the company has already hired several new employees with the series A proceeds, bringing the team to 14 people—of which seven are Ph.D.-level scientists. That headcount should swell to 25 by the end of 2018, which will likely mean additional laboratory and office space is required.

The series A takes Locus’ total fundraising to $21 million to date and will allow the company to “enter strategic partnering discussions from a position of financial strength,” according to Garofolo, who is hoping to sign the first deals within the next 12 to 18 months.

Another CRISPR specialist with antimicrobial drugs in its sights, Eligo Bioscience, raised $20 million in a series A in September but is focusing on the Cas9 system.