RESEARCH TRIANGLE PARK – Morrisville-based Locus Biosciences just got a $77 million shot in the arm to develop its new antibacterial therapy to treat E. coli bacteria-causing recurrent urinary tract infections (UTIs).
Locus said it has signed a contract with the Biomedical Advanced Research and Development Authority (BARDA) to co-fund the development of LBP-EC01. It’s a CRISPR Cas3-enhanced bacteriophage product.
BARDA is a unit of the U.S. Department of Health and Human Services. It will provide up to about half of the $144 million needed to support Phase 2 and Phase 3 clinical trials and other activities required for U.S. Food and Drug Administration approval. BARDA also brings a lot of antibacterial product experience to the table.
Locus will control the development and commercialization of LBP-EC01.
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Close to 150 million people worldwide suffer from UTIs each year, with about 80% of those infections caused by E. coli. That includes difficult-to-treat strains that are resistant to commonly used antibiotics.
“We are incredibly pleased to partner with BARDA to advance our lead asset into advanced clinical trials,” said Locus CEO Paul Garofolo. “Not only will BARDA provide critical expertise to support the program through the later stages of clinical development, but its support dramatically increases the profile of precision medicine to address the emerging worldwide public health crisis around antibiotic resistant infections.”
Using CRISPR-Cas3 to attack bacteria
The company, a North Carolina State University spinout, specializes in Type 1 CRISPR-Cas3-enhanced bacteriophage therapeutics. It starts with naturally occurring bacteriophage (phage) viruses that infect and replicate within bacteria. Then Locus increases their ability to kill the germs by arming them with CRISPR-Cas3.
CRISPR – the acronym for Clustered Regularly Interspaced Short Palindromic Repeats – is a naturally occurring immune system that bacteria use to fight infection.
In recent years, scientists have adopted one form of CRISPR – Cas9 – to edit DNA at specific places in the genome. Locus’s introduction of CRISPR-Cas3 is important because, unlike Cas9, it shreds the target DNA, leading to cell death. That helps destroy residual bacteria that could otherwise survive infection by the bacteriophage.
The beauty of the Locus technology is twofold. It’s a powerful tool to fight infections. Yet it’s believed to be safe for healthy cells because a phage only binds to and kills specific germs.
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Phages have been used to fight bacteria since the early 20th century. But they usually weren’t effective enough on their own to treat serious infections. So the approach was relegated to the back burner with the successful introduction of antibiotics.
Now antibiotic resistance and the emergence of superbugs – along with the booming field of research that connects particular bacteria with diseases and the advancement of CRISPR technology – have caused scientists to take a fresh look.
And that’s the area where Locus excels.
Locus founded with help from NC Biotech
The company was founded in 2015 with the help of a $75,000 Company Inception Loan from the North Carolina Biotechnology Center. The Center followed up with a $250,000 Small Business Research Loan. And three of the four scientific founders of Locus also have received NC Biotech grants totaling more than $300,000.