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Cyntellect Laser-Based Assay System Shows Potential for RNAi Compound Transfection

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In trying to overcome RNAi’s delivery hurdle, many researchers are experimenting with viral vectors, liposomal formulations, hydrodynamic administration, and the like. But one privately held company, Cyntellect, has taken a different tack: the company’s platform uses lasers to temporarily permeablize cells for transfection. Although the technology is not commercially available, Cyntellect has already signed several deals to pilot test the technology for interested parties, one of which is to purchase a prototype of the platform this year.

Cyntellect, a subsidiary of San Diego-based Oncosis, calls its technology LEAP, or laser-enabling analysis and processing. Fred Koller, COO and vice president of R&D for both Oncosis and Cyntellect, told RNAi News that LEAP is designed to perform high-speed image-based cell assays. The system’s use of lasers that can permeabilize cells for about 20 to 30 seconds allows for the transfection of a variety of molecules, including nucleic acids such as siRNAs.

Using a process called Opto-Injection (for which the company has filed a patent application, number 20020076744), Cyntellect has successfully permeabilized cells, injected fluorescently tagged siRNAs into them, and conducted image analysis several minutes later, Koller said. The system, he added, also allows for the use of lasers to destroy untransfected cells, so “you can basically guarantee yourself a very pure population of transfected cells at the end of any process.”

The road to commercializing LEAP, Koller said, first involves the pilot programs. Each is between three and nine months in duration, but with the earliest signed in July, none is nearing completion, he noted. The company has ongoing collaborations with pharmaceutical firms. These projects involve Cyntellect performing work in-house, then showing the results to collaborators who may choose to purchase a beta version of the platform.

Purchasers of a LEAP prototype will provide Cyntellect with certain data from their experiments that will help in making improvements for a commercial version of the system. Once that final version is available, users of the beta platform will receive an upgrade, Koller said. He added that Cyntellect will retain the right to assays developed on the beta platform.

Cyntellect is also in talks with academic institutions regarding purchase of beta versions of the LEAP system, Koller said, and one investigator at the University of Texas Medical Branch at Galveston, James Leary, has already secured a government grant that will be used to acquire the platform before year-end.

In addition to Leary’s lab, Koller said that the company hopes to get two more beta systems into the hands of external labs through deals with academic or industry organizations within the next four to six months. Koller noted that several of the parties Cyntellect is in discussions with over possibly purchasing a prototype system “have RNAi as a component” of their work, primarily RNAi for research and development purposes. He also said that Cyntellect has been approached about using the technology for RNAi-based drugs by “the companies that you’d think of in the space.

“The technology does deliver the RNA to the cells. So if you’re looking at doing this in blood cells or some other cell that can be taken out of the body and put back, that’s do-able,” he said. “It’s not going to work for a liver treatment, but if it’s a stem cell or blood cell therapy … that’s feasible,” Koller said.

“We’re talking to a few of the RNAi [industry] players right now, but they’re fairly early discussions,” he said, declining to be more specific. He added that such deals are not something that Cyntellect is “aggressively pursuing.”

Whoever the customers for the beta version of the LEAP platform turn out to be, their feedback is expected to help Cyntellect to launch a full-fledged commercial version of the system in early 2005.

Whoever the customers for the beta version of the LEAP platform turn out to be, their feedback is expected to help Cyntellect to launch a full-fledged commercial version of the system in early 2005.

The cost of a commercial LEAP system has not been established, Koller said, but he noted that “it’s going to be in line with other high-end instruments in the marketplace. It’s going to be comparable to the high-end imagers that are out there, products like [Amersham’s] INCell 3000, the Evotec Opera.

“It will mostly end up being in the same price point as those types of instruments,” he said, which “range from $500,000 up to $1 million.”

The price of beta versions, he added, would be negotiable depending on the resources a user was willing to contribute to Cyntellect’s effort to further develop the system.

—DM