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After Publication of Data on siRNA Delivery, Leonardo Biosystems Sets Sights on Partnerships

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By Doug Macron

Just weeks after the publication of data demonstrating the potential of its proprietary delivery system in RNAi, Leonardo Biosystems has begun ramping up operations with the goal of securing industry partnerships around the technology, a company official told RNAi News this week.

"The theory of the business is to be a drug-delivery company and a source of solutions for the broad intravenous drug-delivery space," Leonardo CEO Bruce Given said. Leonardo is "trying to become a partner of choice for [companies] that need solutions for the various challenges they face with their products."

And while the company may at some point down the road dip its toes into the drug-development waters, "initially, I don't see us having any in-house discovery efforts," he added. "I don't see us taking any home-grown products all the way through" to commercialization.

Leonardo was co-founded by Arrowhead Research CEO Christopher Anzalone and University of Texas Health Science Center researcher Mauro Ferrari to commercialize the delivery technology, which was developed in Ferrari's lab. Arrowhead holds a minority stake in the company.

The technology involves a so-called multi-stage system comprising mesoporous silicon particles containing neutral nanoliposomes carrying a drug payload. Earlier this month, Ferrari and colleagues reported in Cancer Research how the system was able to deliver siRNAs targeting the oncoprotein EphA2 into two mouse models of ovarian cancer.

The team found that a single intravenous injection of the drug was able to silence the EphA2 gene for at least three weeks and reduce tumor burden, angiogenesis, and cell proliferation without showing signs of toxicity.

In an interview with RNAi News, Ferrari pointed out that the tumor shrinkage was obtained "without concurrent chemotherapy … [which] is a major achievement" (RNAi News 5/20/2010).

According to Given, these data were "a compelling bit of proof of concept," and will support the company's efforts to find a partner within the RNAi space. But Leonardo has designs on applying its delivery technology to other areas, as well, and "we would like to broaden that proof of concept into other areas [such as] chemical entities."

For instance, Given said that the technology has the potential to improve upon existing chemotherapeutics that, while effective, are highly toxic, by limiting their impact on healthy tissue and increasing the amount of drug that gets to tumors.

Still, the technology's promise for therapeutic RNAi applications "is huge," and remains a key focus for Leonardo, as are microRNAs and other small RNAs, he noted. "I think that whole [small RNA] area is in need of elegant delivery solutions, and I would expect that we could be part of the solution for a great number of those technologies."

The delivery of RNAi drugs using the multi-stage system has so much potential, in fact, that Given suggested that Leonardo could possibly participate in the development of the siRNA-based therapeutic detailed in Ferrari's Cancer Research paper.

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Still, "the ultimate goal is to be a partner and have [a drug's] development aspects [and] the clinical programs being run by others," he pointed out.

Thus far, Leonardo, which has just two employees, has been run as a "virtual company … on individual angel investments," Given said. But with the expected receipt of grant funding through the Texas Emerging Technology Fund, the firm is "getting off the ground … [and] moving into a more active stage."

A major part of that effort will involve the hiring of scientific staffers to support the company's "solidifying of the technology base so that we can go out there and seek partnerships," he explained.

Thus far, Leonardo has not been actively pursuing deals because, according to Given, "there's always risk if you go out too early. The time to go looking for partners is when you have a clear story to tell them and a clear offering for them."

To do so, Leonardo is planning to conduct work to "understand how to tune the [delivery] particles to get the desired specification," Given said. "In certain applications, you might want these particles to stay in the circulation and release [drug] over several days … [or] several weeks. In [other] applications, you might want the particles to go to specific places … such as slow-moving tumor vasculature, for instance.

"There is also the possibility of decorating these particles with various agents that might cause them to adhere, for instance, to vasculature that is expressing certain epitopes and cause the particles to accumulate in one place," he said.

On top of this basic scientific work are more "mundane things," such as ensuring that the company can meet chemistry, manufacturing, and control requirements "so that we can be ready from a regulatory perspective to work with our partners," he added. "So there's basic, foundational work to be done that will put the company on a firm footing going forward."

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