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Isis, Rosetta Ink Deal to Develop miRNA-Targeting Drugs and Ambion Unveils miRNA Analysis Service

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Isis, Rosetta Ink Deal to Develop miRNA-Targeting Drugs …

Isis Pharmaceuticals and Rosetta Genomics announced this week that they have established a partnership to discover and develop antisense drugs that regulate microRNAs for the treatment of hepatocellular carcinoma.

The deal brings together Rosetta's miRNA expertise and intellectual property with Isis' experience in oligonucleotide chemistry and antisense research and development.

"Think of them as the front end, [finding] the appropriate microRNA [and identifying] the targets … and think of us as providing the … drug discovery and development in antisense," Stanley Crooke, founder, chairman, and CEO of Isis, told RNAi News.

Terms of the deal were not disclosed, but Crooke said that the arrangement has a two-year term, and calls for Rosetta to identify miRNAs implicated in hepatocellular carcinoma. The companies then have the option to jointly discover and develop antisense drugs against the targets using Isis' technology, he said.

Should the companies choose not to collaborate on a particular drug, either one may license it for in-house development, which is "a high-quality problem to deal with in the future," he said.

Crooke declined to provide a timeline for when he expects drug candidates to result from the deal with Rosetta, but said that "we think we can make very rapid progress given what they know and what we know. … We believe two years is sufficient to do the research to identify attractive candidates."


"We … view antisense as the optimal platform for inhibiting miRNA function."

Late last year, Rosetta Chairman and CEO Isaac Bentwich told RNAi News that his company developed a bioinformatics platform that has been used to scan the human genome and identify miRNAs (see RNAi News, 11/4/2005). He said at the time that Rosetta had confirmed about 200, and expected to identify as many as 1,000. He stressed at the time that he anticipates Rosetta's intellectual property estate will give it "ownership of 50 to 80 percent of the microRNAs in the world."

Bentwich also told RNAi News that while Rosetta was planning on developing drugs targeting miRNAs, the company intended to "collaborate and use whatever is the best technology" to target the small RNAs. For Rosetta, it seems that technology is antisense.

"We … view antisense as the optimal platform for inhibiting miRNA function," Amir Avniel, president of Rosetta, noted in a statement this week.

Should antisense prove to be an effective means of silencing miRNAs, the deal with Rosetta could breathe new life into Isis, which has faced numerous failures in developing antisense drugs. Thus far, Isis has only brought one to market, Vitravene, which is used to treat cytomegalovirus retinitis in AIDS patients — a relatively small patient population.

However, earlier this month Isis published data in Cell Metabolism demonstrating that a 2'-O-methoxyethyl phosphorothioate antisense oligonucleotide could be used to inhibit the expression of the liver-specific miRNA miR-122 in mice — indicating that the technology still has promise.

According to Isis, the "inhibition of miR-122 in normal and high fat-fed mice resulted in a significant improvement in numerous metabolic and cardiovascular risk factors as evidenced by reduced plasma cholesterol levels, increased hepatic fatty-acid oxidation, decreased hepatic fatty-acid and cholesterol synthesis rates and reduced fat in the liver. These results implicate miR-122 as a key regulator of cholesterol and fatty-acid metabolism in the adult liver and suggest that miR-122 may be an attractive therapeutic target for cardiovascular and metabolic diseases."

… and Ambion Unveils miRNA Analysis Service

Last week, Ambion Services officially unveiled a new expression profiling service specifically for miRNA analysis.

According to Scott Hunicke-Smith, vice president of Ambion Services, the service runs customers' samples of total RNA, which must be purified so as to preserve the miRNAs, through the company's mirVana bioarray to yield "very reproducible microRNA profiles." He added that "we can also go backward to tissues and blood, and do that same profiling."

Hunicke-Smith said that applications of the service fall into two categories: hypothesis-driven and hypothesis-generating experiments. "The hypothesis-driven experiment [involves] someone with an in vitro model and they're trying to determine what a compound or some environmental factor is doing to the microRNA profiles. That is much more along the lines of what is now a classic microarray experiment," he said.

"The other category is tissue-based, which is the hypothesis-generating [experiment]," he said, wherein a researcher may have "interesting tissues representing normal state and disease state, and [they] want to understand if microRNAs can be implicated in the underlying condition. So we'd do profiling of a statistically significant number of the normal and disease, and help them look for patterns of change … [or] biomarkers."

Hunicke-Smith noted that Ambion sees discovery groups at biotechnology and pharmaceutical companies and clinically oriented academics as the key customers for the new service.

He noted that Ambion beta tested the service in the fourth quarter of last year, and began taking on new customers early this year, "but we haven't announced it broadly until just now."

Early this year, Ambion announced that it signed a deal to sell its research products division to Applied Biosystems for $273 million in cash (see RNAi News, 1/5/2006). The deal does not include Ambion's services and diagnostics divisions, which are to be spun out into a new firm called Asuragen once the acquisition closes.

— Doug Macron ([email protected])

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