RESEARCH TRIANGLE PARK – Global pharmaceutical giant Eli Lilly and Company is interested in Durham-based Precision Biosciences’ genome editing technology and it’s putting its money where its interest is.

The two companies announced last week a research collaboration and exclusive license agreement that could potentially be worth over a half billion dollars. Lilly will use Precision’s proprietary ARCUS genome editing platform to research and develop therapies for genetic disorders. It will start with Duchenne muscular dystrophy (DMD) and two other undisclosed gene targets.

Under the terms of the agreement, Precision will receive an upfront cash payment of $100 million and an equity investment by Lilly of $35 million in Precision’s common stock. Precision also is eligible to receive up to $420 million in development and commercialization milestone payments per product, along with tiered royalties, if Lilly is successful in launching a new therapy.

Precision Bio photo

Precision Bio facility

Precision will be in charge of pre-clinical research and Investigational New Drug-enabling activities. Lilly will handle clinical development and commercialization. Lilly will have the right to select up to three additional gene targets as part of the collaboration. And Precision can co-fund clinical development of one product in exchange for increased royalties on that product’s sales, according to the agreement.

This is the latest in a series of blockbuster announcements by pharmaceutical companies from throughout the world that are being drawn to the Research Triangle area as a global magnet for cutting-edge cell and gene therapies. Lilly announced in January it is building its first North Carolina manufacturing facility in the Durham County portion of Research Triangle Park, with plans to invest $474 million and create 462 new jobs.

“We look forward to working with Lilly to leverage our deep understanding of in vivo gene editing experience with ARCUS to develop new therapies, including a potentially transformative treatment for Duchenne muscular dystrophy,” said Derek Jantz, chief scientific officer and co-founder of Precision. “Collaborating with Lilly…will help us accelerate our work aimed to solve genetic diseases with unique editing challenges.”

The ARCUS gene editing technology was discovered and developed by Precision scientists. It uses DNA-cutting enzymes – or nucleases – to insert, remove or repair the DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in a certain type of algae.

“Gene-edited therapies are emerging as a promising approach to help patients afflicted with genetic conditions, said Ruth Gimeno, Ph.D., vice president of diabetes and metabolic research at Lilly. “We look forward to working closely with Precision’s scientific team and leveraging their platform to develop and deliver breakthrough medicines for untreated genetic disorders.”

About Precision

Precision is a clinical-stage biotechnology company that was spun out of Duke University in 2006 and went public in March 2019. Its shares are listed on the Nasdaq Global Select Market under the ticker symbol “DTIL,” shorthand for the company’s marketing tagline, “Dedicated to improving life.”

Lilly signs gene editing deal worth up to $555M with Durham’s Precision BioSciences

Precision has close to 225 employees. Its pipeline includes several “off-the-shelf” CAR T immunotherapy clinical and in vivo gene correction therapy candidates. They are targeted at curing genetic and infectious diseases for which no adequate treatments exist.

In July 2019 Precision opened its Manufacturing Center for Advanced Therapeutics, the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

“Given the potential output of our platform, we’ve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients,” CEO Matthew Kane said when the facility opened. “In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.”

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

(C) N.C. Biotech Center

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