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RNAi Roundup: Benitec Heads to California, Plans IPO; NYAS To Hold RNAi Symposia; Qiagen in Deals with Novartis, Amaxa

NEW YORK, Aug. 8 (GenomeWeb News) - Less than four months ago, when Benitec exclusively licensed to Promega the rights to use its ddRNAi (DNA-directed RNAi) technology, the Australian company took a major step in its strategy to set up shop in the US as a therapeutics developer, executives from the companies indicated this week.


Now, it appears that the deal is ready to bear fruit and that Benitec is poised to relocate, as well as float its stock on the NASDAQ.

"We have to be where the market is," Benitec Chairman and CEO John McKinley told GenomeWeb, noting that, although a small portion of the company's activities may continue in Australia, the majority of all RNAi activity is occurring in the US.

Benitec intends to build facilities to house its corporate and commercial activities in California, possibly in San Francisco or San Diego, and expects to begin "laying down the groundwork" for this effort in the next three months, McKinley said.  Then, in the summer of 2004, the company intends to proceed with the initial public offering.

To ensure the success of its transformation from a small Queensland, Australia-based firm with little more than a handful of patents to a full-fledged drug developer in the US, Benitec wants to establish a revenue stream that will help offset the cost of its research activities, as well as help build a portfolio of independent research validating its claim that ddRNAi really works.

Key to the first goal is the Promega deal.

In early April, Madison, Wis.-based Promega picked up the global rights to use the ddRNAi technology in areas outside human therapeutics.  Under the arrangement, Benitec received an upfront fee and stands to be paid royalties on sales of the laboratory and analytic research products developed by Promega using the ddRNAi technology, in addition to a portion of the revenues Promega derives through sub-licenses.

Richard Schifreen, Promega's director of technology and market development,   told GenomeWeb this week that his company will have ddRNAi products ready for the market this fall.  Meanwhile, plans are underway at both companies to begin seeking out licensees for the technology within the next few months, and Promega has already included on its website a page ( ) detailing the patents on the technology and providing a link through which licensing information can be requested.  

But even without an active sales effort, McKinley and Schifreen said that the companies have been receiving inquiries about licenses to the technology, and that discussions are underway with a number of parties, including RNAi start-up Nucleonics, which is working on a technology similar to ddRNAi that uses what the company has termed expressed interfering RNA, or eiRNA.

As for Benitec's efforts to make believers out of those that may look to ddRNAi with a skeptical eye, McKinley said that the company is currently pursuing collaborations with medical research and academic institutions to conduct experiments with the technology.  

As the excitement surrounding RNAi continues to grow, an increasing number of conferences dedicated to the field have been popping up.  Now, the New York Academy of Sciences has joined in, planning to hold later this year in New York City the first in an ongoing series of symposia designed to bring together RNAi researchers to discuss their work.

Rashid Shaikh, director of programs as NYAS, told GenomeWeb that the institution has been holding similar meetings in other scientific areas, such as structural biology, bioinformatics, and drug discovery, for years. "This year we are starting three or four new ones, and RNAi is one of them."

He said that NYAS decided several months ago to expand its symposium programs to include other fields and began putting together a list of possibilities.  "When we started talking to people both in academia and in some companies, there was a lot of interest in RNAi."

One of those academics is Tom Tuschl, now a Rockefeller University Associate Professor, who was part of the team that demonstrated early on that RNAi could be introduced into mammalian cells without inducing a fatal interferon response. Tuschl and RNAi researcher Gregory Hannon of Cold Spring Harbor Laboratory, are acting as informal scientific advisors to Shaikh, and helping to organize the events.

Tuschl told GenomeWeb that the symposiums are expected to last about half a day each, and include presentations from two high-profile names in RNAi - one from within the New York area and one from elsewhere - followed by talks from three to four postdocs or graduate students selected through a competition.

"The idea is for people to be talking about work that they are doing, and hopefully a lot of this work will not be published yet so that it's really new and exciting for people to hear," Shaikh noted.

Pointing out that it can be difficult to attract attendees for conferences in New York, given the number of scientific meetings that occur at any one time here, Tuschl said he wants the symposia to be of sufficient size and scope to warrant someone choosing to attend NYAS.

"There is always five meetings you can go to at a time [in New York]," he said.  "So you want to provide sufficient time and a sufficiently large program...and at the end there will be extra time for socializing if you want to establish collaborations or talk to people."

According to Tuschl, the symposia are expected to deal with three main topics: the application of RNAi as a tool for studying gene function, the mechanism of RNA silencing, and chromatin modifications guided by small RNAs.

The plan is to hold each at a different location throughout New York City where RNAi research is being conducted, with the first slated to occur at Rockefeller in early November.

Although the roster of presenters for this meeting has not been finalized, NYAS has already contacted Phillip Sharp, a Nobel Laureate and MIT Institute Professor, who worked with Tuschl on researching RNAi in mammals, and Christophe Echeverri, founder and CEO of Cenix BioScience about attending as keynote speakers.

Qiagen has significantly grown its RNAi operations since first picking up siRNA technology through its April 2002 acquisition of Xeragon, and a flurry of activity this week appears to indicate that this trend may continue.

When Qiagen bought Xeragon for $8 million in stock, it was following an acquisition strategy that chief financial officer Peer Schatz characterized during the company's second quarter conference call this week as taking "very small pieces of technology that give us a very critical advantage in rapid growth areas or very important technology areas for Qiagen's core competencies."

The Xeragon deal led to Qiagen's introduction of a number of siRNA products, including the recently introduced "4-for-Silencing" custom siRNA duplexes, and the striking of an siRNA supply deal with Intradigm - both of which contributed to the Dutch firm's 11 percent rise in second quarter revenues from oligos.

This week, Qiagen also announced that it had formed two separate deals for its siRNA technology with Swiss drug giant Novartis and private German biotech Amaxa, respectively.

Under the first deal, Qiagen will provide Novartis with a genome-wide library of siRNAs for use in its drug discovery efforts.

Patrick Weiss, vice president of gene silencing at Qiagen and founder of Xeragon, told GenomeWeb that while the collaboration is a "big deal" for Qiagen from a financial point of view (he declined to comment on the financial details beyond this), the real importance of the arrangement lies in that, as far as he knows, this is the first time a company will create a siRNA library that covers the entire human genome, and that Novartis is "probably a year in advance of the other pharmas" in its plans to use the far-reaching oligo database.

Weiss said that Qiagen expects to have the siRNA library completed for Novartis by the end of October.

Under the second partnership, Qiagen has agreed to market Amaxa's Nucleofector technology - used to transfect siRNA oligos and siRNA vectors into primary cells, hard-to-transfect cells, and common cell lines - with its own oligos.

Specific terms of this deal were not disclosed..

Allele, of San Diego, said this week that it has received two six-month SBIR grants, for $100,000 each, from the NIH to support its development of its RNAi technology.  The company is also planning to introduce a new dsRNA transfection peptide shortly.  

The first grant will fund development of Allele's RNAi microarray technology, called Cell Array, which is essentially a system for growing cells that have been transfected with DNA constructs that encode siRNA.

The second grant, the company said, will support the second-generation development of a peptide the company believes can transfect cells with RNAi-based polynucleotides 10 and 100 times more efficiently than cationic lipids.

Jiwu Wang, president of Allele, told GenomeWeb that his company has been working on the peptide technology for about half a year and it has proven more successful than previously expected. As such, Allele has sent out beta versions to different research facilities for testing and expects to have a final product ready for commercialization in about a month.

He said that Allele is in discussions with Promega - which already distributes its LineSilence DNA oligo-based gene target screening system under the name SilentGene - about promoting the peptide.  However, he added that Allele would prefer if possible to handle the marketing effort itself.

The two newest grants come from the National Cancer Institute and the National Institute of General Medical Sciences. The company had previously received three other SBIR grants, altogether worth $746, 469.

In other news, Dharmacon said on Aug. 5 that it has received a new US patent protecting the firm's core synthetic RNA oligo production technology.

The patent, no. 6,590,093, covers "novel orthoesters...which can be used as a 2'-hydroxyl protecting groups or 2'-modification in the synthesis of polymers containing ribonucleic acid nucleotides."

The orthoesters, said the Lafayette, Colo.-based company, protect the RNA and thereby  minimize degradation during handling, purification, analysis, and shipping.

Nucleonics, of Malvern, Pa., said that it has named Robert Towarnicki as its president and CEO.

Towarnicki was formerly the president and CEO of Cell Pathways, which was acquired by OSI Pharmaceuticals, a cancer therapy developer, in June this year, and was COO of Integra LifeSciences before that.

"Robert Towarnicki has shown considerable skill over the years in building emerging companies, first at Integra LifeSciences and then at Cell Pathways," Maxine Gowen, Nucleonics board member, said in a statement . "His experience should serve Nucleonics well as the company advances over the next 18 months from preclinical development to the clinical testing of its novel eiRNA-based therapeutics for viral and other diseases."

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