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Horizon Racks Up More Academic Research, Licensing Deals with Torino, Newcastle Universities

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By Ben Butkus

This article was originally posted on Aug. 11.

Horizon Discovery this month inked collaborative research and licensing deals with a pair of European universities as part of its ongoing strategy to build out its personalized medicine drug-discovery capabilities through academic partnerships.

In the first deal, announced last week, Horizon said that it has secured an exclusive, worldwide license from the University of Torino relating to the use of human isogenic cell lines containing patient-relevant genetic mutations to predict drug response in individual cancer patient populations.

And this week, Horizon said that it has signed a research collaboration with scientists funded by Cancer Research UK and located at Newcastle University to identify biomarkers that can be used in non-invasive cancer-imaging applications.

In addition, later this year the company plans to launch an open-source consortium of "top academic institutions and big pharmas" in the cancer field, a research partnership that is expected to leverage and expand Horizon's panel of isogenic cell lines for drug discovery, a company official said this week.

Horizon, based in Cambridge, UK, on the campus of the Babraham Research Institute, has additional research laboratories in Torino, Italy, and has consorted heavily with a number of universities and non-profit research institutes since it was founded in June 2007.

At the core of its academic-partnership strategy is an adeno-associated, viral-based, gene-engineering technology platform known as Genesis, to which the company has a worldwide exclusive license from the University of Washington.

The Genesis technology, which uses a viral vector to insert specific nucleic acid sequences into human cells, allows researchers to induce homologous recombination at a much higher efficiency — anywhere from 100 to 1,000-fold — than DNA plasmid technology, according to the company.

Using this technology, the company is able to generate human isogenic cell lines — normal and mutated cancer cells that can be used to discover drugs tailored to the specific mutations that occur in the cells of cancer patients. Horizon also has an exclusive license from Johns Hopkins University covering the sale and use of its inventory of isogenic disease models generated by JHU researchers with the Genesis technology over the previous decade.

The company develops its own isogenic cell lines, which it then sells to drug-discovery or pharmaceutical companies, or makes the basis of a drug-discovery service offering for such firms.

However, it also taps into academic institutions as much as possible to develop a larger repertoire of cell lines than it could on its own as well as new applications for the cells and the Genesis technology.

"We have nearly 100 peer-reviewed publications on this technology," Darrin Disley, Horizon's commercial director, told BTW this week. "Nearly everything in our company originated from academia. It's one reason we're in such a strong position. If anybody wants to look at the cell lines we're using they can look at publications. It's not like we're trying to convince people about new, unproven technology."

Indeed, Horizon owns the rights to some 150 isogenic cell models, which it markets under the name X-MAN, and approximately 100 of which have been in-licensed from various academic partners, but mostly Johns Hopkins University, with which Horizon has an ongoing commercial partnership.

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Horizon broadly calls its strategy the "Targeting Cancer Consortium," which describes the process of letting academic labs use its Genesis technology at reduced rates, with the understanding that it will have first dibs on any new cell lines or IP that come out of the research. Horizon then exclusively licenses the cell lines or IP back from the university to use in its drug-discovery offerings, for which the school receives an ongoing royalty on sales of the cell lines.

Horizon's most recent agreement under the Targeting Cancer Consortium model was announced in late February and involved the University of Maryland, Baltimore, which granted an exclusive license to the company for isogenic cancer cell lines developed at the school by former JHU postdoc Kurt Bachman (see BTW, 4/1/2009).

The agreements announced this month continue Horizon's academic courtship, but have a slightly different flavor than its Targeting Cancer Consortium model, Disley said.

The University of Torino licensing agreement, for instance, is the result of an ongoing research collaboration with Horizon co-founder Alberto Bardelli, who heads the department of oncological sciences at the university. Disley said that the university has a sub-license to the Genesis technology and manufactures isogenic cell lines exclusively for Horizon. "We do all of the commercial cell line generation, and they work on more of the cutting edge, novel models for new applications," Disley said.

Bardelli's work has specifically focused on mutations of the KRAS gene, which imparts resistance to EGFR-targeted agents in colon cancer. "He's been developing a lot of new applications for how you use gene-engineering technology and the subsequent isogenic cell lines for all kinds of translational medicine applications: modeling patient resistance, how you can reverse that resistance, et cetera," Disley said.

Bardelli recently filed new patent applications related to some of those discoveries, which Horizon exclusively licensed. Under the agreement, Horizon will pay the university undisclosed fees and will assume prosecution and sub-licensing control of the IP, the company said.

Horizon's research collaboration with Newcastle University, meantime, is intended to bolster the company's recent entry into the companion diagnostics space, which it began last month with a partnership with UK-based personalized medicine company DxS. Horizon's foray into companion diagnostics is "initially with DXS, but we'll be working with all kinds of groups on developing standards and controls for these tests," Disley said.

To wit, Horizon will work with Newcastle researchers Herbie Newell and Ross Maxwell, who are funded by Cancer Research UK, Europe's largest cancer research funding agency. Specifically, Newell and Maxwell will attempt to identify genotype-specific biomarkers that can be used in non-invasive cancer imaging, "so clinicians can basically do this without needing to take invasive biopsies," Disley said.

Although Horizon declined to disclose commercial details of the agreement, Disley said that as with its other academic collaborations, Horizon "owns the cell lines [and] modifications to the cell lines, and we have first options to IP." That IP would be negotiated with both Newcastle University and CRUK due to a pre-existing agreement between those entities.

In the fourth quarter of this year, however, Horizon plans to announce a call for project proposals from academic research groups to access the Targeting Cancer Consortium strategy, Disley said.

"This is going to be sponsored by several big pharma companies and top academic institutions in the cancer field," Disley said. "And it's under that model where we enable people with our core platform technology and maybe some cell lines to start with; and they create either new materials or modified materials within a research program, and other IP." He declined to disclose any of the partners at this point.

"It's the same kind of model: We own all the cell models, they get a royalty back, and any new IP we have first option to along with our partners," Disley said, adding that the company hopes to announce the initiative in Q4 and launch it in Q1. "We've got some of these relationships already, but we're going to formalize it with two of the big cancer pharmas," he said.

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