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Horizon Discovery's UMB Cell-Line Deal Latest Example of Its Academic Collaboration Model

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This story has been updated from a previous version to correct factual information about Horizon Discovery's Genesis technology.

British biotech tools company Horizon Discovery has expanded its line of cellular models for cancer by licensing a panel of new patient-relevant human isogenic cell lines from the University of Maryland, Baltimore, the company said last week.

The licensing deal underscores Horizon's academic collaboration strategy, dubbed the "Targeting Cancer Consortium," in which it supplies academic researchers with its cell lines and viral-based gene-engineering platform to create cellular disease models that it then licenses back and sells to pharmaceutical companies for a variety of uses.

"We are quite happy and encourage academic institutes to partner with us and use our platform and cell lines at nominal rates, and then publish [their results]; and then we provide a route to commercialization," Darren Disley, Horizon's commercial director, told BTW last week. "Then we have a pipeline [of products and services] to provide to big pharma."

"Conventionally people think about tech transfer as being very linear, in that there is an invention made using government funding, and that invention is licensed to a company," Libby Hart-Wells, senior director of UMB's Office of Commercial Ventures and Intellectual Property, told BTW.

"Nowadays, there are all kinds of complex and creative collaborations out there," Hart-Wells added. "It has required our office to adapt away from that very linear thinking and encourage these types of collaborations. They're equally fruitful for the public at large."

Under the terms of its agreement with UMB, Horizon, based in Cambridge, UK, received an exclusive, worldwide license to isogenic cell lines that UMB scientists developed using Horizon's adeno-associated viral-based gene-engineering technology platform.

That platform, called Genesis and exclusively licensed from the University of Washington, allows researchers to induce homologous recombination in cells at "extremely high efficiencies" — anywhere from 100 to 1,000 times more efficiency than DNA plasmid technology, Disley said

The technology uses a viral vector to insert a sequence of 4.6 kilobases paired to any gene in a human cell, Disley added.

"It can ensure that the insertion event only occurs at one single gene loci," Disley said. "It's exquisitely sensitive and precise at targeting this. This allows you to take normal human cells, introduce a single point mutation, and start building up cancer models because you have the normal cell and the cancerous cell."

In exchange for the cell lines, UMB received an undisclosed up-front payment and is entitled to ongoing royalties on sales of products related to the cell lines. Although the specific royalty rate was not disclosed, Disley told BTW that it was in the "low double digits," which is "reasonably large" for a research tool license.

The license agreement is initially for seven years, with an option to renew at the end of that period, Horizon said.

Bal'mer Connection

Horizon will add the new cell lines to its stable of more than 150 cell models, which it markets under the brand name X-MAN. Approximately 100 of those cell lines have been in-licensed from academic institutions, while Horizon developed the rest, Disley said.

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Horizon has licensed the majority of those cell models from Johns Hopkins University, particularly from laboratories associated with the school's Kimmel Cancer Center.

Horizon has also licensed from JHU methods for using the adeno-associated viral vector technology to create cellular disease models, which were developed by Horizon CEO and principal founder Chris Torrance, a former researcher in the oncology lab of Johns Hopkins researcher Bert Vogelstein; and Alberto Bardelli, a researcher at the Institute for Cancer Research and Treatment in Turin, Italy.

Torrance's familiarity with Johns Hopkins and Vogelstein's lab in particular helped facilitate a professional relationship with Kurt Bachman, another former Vogelstein postdoc. Bachman eventually took a visiting faculty position with UMB, where he used the Genesis technology to develop the new cellular disease models that Horizon licensed from UMB.

UMB's Hart-Wells said that Bachman, who has since moved on to the private sector, was crucial to facilitating the agreement.

"We began working with Horizon through a good working relationship with [Bachman], who was a part of our marketing team," Hart-Wells said. "Most of the time when we do licenses, it's really to the credit of the faculty innovator being part of that process."

The bulk of UMB's tech-transfer activity falls under the broad umbrella of life sciences, Hart-Wells said. And although licensing deals for biological research tools might not provide as lucrative a return for the school as, say, a potential lead compound for a pharmaceutical product, the importance of such deals is increasing, she said.

"I think our office has in the last couple of years dug in on these biological materials that are assets, whether or not they're developed in collaboration with other people," Hart-Wells said. "This is a healthy way to sustain the scientific enterprise."

Horizon and UMB could see royalties from the licensing deal through one of three main commercial routes for the X-MAN cell models. The first is in drug discovery, where Horizon sells either cell lines or research services based on them to pharma companies to identify drugs for particular genetic defects.

"Traditionally cancer drugs have been administered in a one-size-fits-all approach," Disley said. "This allows pharma companies to target particular defects. For instance, people with particular mutations cannot be administered certain drugs for colorectal cancer."

The second commercial area is using the cell models to validate screening tests for particular mutations in patients' cells, Disley explained. For instance, he said that if one wanted to screen a patient for a particular mutation that was incompatible with certain colorectal cancer drugs, "you could validate a screening test for that on these cell lines [because] you can control the amount of mutation."

These two applications are Horizon's bread and butter, for the time being, but the company is also hoping to use the X-MAN technology to break into the bioproduction market — the third commercial route — "because it's a genetic engineering technology that is exquisitely precise, [so] you could create cells for bioproduction purposes," Disley said.

He added that Horizon will likely announce a collaboration with a "big player in the bioproduction field" in the coming weeks, but declined to elaborate.

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