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RNAi Roundup: Scaringe, Back Out Front, Discusses Dharmacon s Abbott Deal and Its Strategy; Alnylam Licenses Polymer Drug Delivery IP and Hires Vincent Miles; And Others

NEW YORK, Aug. 1 - Dharmacon's new deal to provide Abbott with a library of short-interfering RNAs for drug target genes is not just another gold star for the company, according to Stephen Scaringe, who has recently taken a new title of Chief Scientific officer and co-chairman.

 

The collaboration, announced Tuesday, fits into Dharmacon's "master program" of developing its siGene list, a library of siRNA pools for common genes that it is selling on its website, Scaringe said. With the work performed under this agreement, "there's a lot of information going back and forth that is helping us overall design and build the siGene list library." This is also true, he said, with the company's collaboration, announced in June, to provide 600 pools of siRNA for Exelixis. However, this does not mean that the pools of siRNA that the company offers on its list to other customers will all be "the same thing" as the ones made for Abbott and Exelixis. "Abbot has particular specific needs and they're definitely getting some uniqueness and advantages with the library they're purchasing from us," he said.  The company is working on "similar deals" with other pharmas and biotech companies "down the road," Scaringe added.

 

Overall, this move to make libraries of siRNA is part of the company's strategy to edge out the competition by becoming "an application developer," rather than just a provider of synthetic siRNA, Scaringe said. He likened the situation to the early days of Sanger sequencing, when a provider of DNA oligo primers could either "sit there and say 'oh, this is cool, it means I get to make more DNA oligos'," or could ask itself how to expand its business to advance sequencing technology."That's what we're doing by analogy with siRNAs. What can we do to make siRNA an incredibly more powerful technology provide that leadership?," Scaringe said. "This platform, the smart pool technologies, the siGene list platform is what we're working to do to provide that leadership."

 

Speaking of leadership, Scaringe's own leadership role at Dharmacon has been in flux recently. Until June, he was CEO. Then, the company's executive vice president of R&D Bill Marshall came to the forefront, and Scaringe disappeared from view amid rumors that he was leaving.The company would only say they were in negotiations with Scaringe about his future role there, while Scaringe denied to GenomeWeb that he was exiting the company. This week, folded into the Abbott press release, Scaringe reappeared with his new title. The company, and Scaringe, would not discuss the matter further, but two things are sure: the rumors of his demise at the company have been greatly exaggerated and he is back out front.

 


In the RNAi therapeutic arena, Alnylam continued the move to expand its management team and its IP position this week, announcing the licensing of two groups of patent applications and the hiring of Vincent Miles as senior vice president of business development.

 

The IP includes an exclusive therapeutic license to US Patent Application No. 20030027783, "Inhibiting gene expression with dsRNA," which describes inventions by Magdalena Zernicka-Goetz and colleagues at University of Cambridge that concern "the specific inhibition of gene expression in mammals by bringing the target into contact with double stranded RNA (dsRNA)," according to the application.

 

Alnylam licensed the application, which was published February 6, from Cancer Research Technology of the UK, a licensing arm of Cancer Research UK and other cancer research institutes.

 

The company also obtained from MIT an exclusive therapeutic license for patent applications for drug-delivery inventions by MIT scientist Robert Langer that can be applied to RNAi therapeutics. These five applications include ones that "involve polymer microspheres," Langer wrote in an e-mail to GenomeWeb. "Other[s] are polymers that behave like viruses without the toxicity and effectively get nucleic acids into cells."

 

This latter group of licenses seems slated to help Alnylam meet the challenge of drug delivery with polymer delivery vehicles. But Miles cautioned against reading it as a sign that Alnylam will definitely choose polymers as the preferred method of drug delivery. Rather, he said, it's just a piece in the company's overall strategy to "build a strong portfolio of IP around siRNAs and their therapeutic use." Miles also emphasized that these licensing moves were part of the company's partnering strategy. "It gives the partner comfort that as you are engaged in discussions with them, that among other reasons, the IP position is a good reason to be working with Alnylam."

 

Miles, who joined Alnylam July 21st from Millennium Pharmaceuticals, has already hit the road on a partnership prospecting tour, talking to pharmaceutical and biotech companies that are interested in the opportunity that RNAi therapeutics provide. (He spoke to GenomeWeb from undisclosed location in California.) 

 

While Miles said that the company was exploring the full range of financial "moving parts" in these potential partnerships, from up-front payments to structured milestone payments, royalties, and other terms, he did note that the kind of deal where a company like Anlylam hands over a clinical or preclinical entity to a pharmaceutical company "is not the best thing for the biotech company," given that the original company then loses all control over whether the project can continue moving forward into clinical development. Instead, he is looking to craft relationships that help Alnylam meet its goals of "having a significant pipeline of RNA therapeutics and becoming an independent product company."

 

Boston-area biotechs and pharma companies would seem an obvious choice for partnership with Alnylam, and Miles did acknowledge that "geography can help" in some instances. But he all-but-ruled out a deal between Alnylam and Millennium, given that Millennium has recently honed its focus toward later-stage clinical development, and Alnylam is in early-stage preclinical development.

 

Miles worked at Millennium Pharmaceuticals for six years, most recently as vice president, business and technology management. He was encouraged to make the leap by Alnylam CEO John Maraganore, who left Millennium last December. But he was also motivated by the "sheer level of interest" in the scientific community for RNA interference technology, and the "vibrant" and "open" corporate culture at Alnylam.

 

"I think the early signs are that we've got the sort of intercommunicative culture where people are sharing information and are very excited about working together to tackle the challenges," he said, "and that reminds me of the pull-together atmosphere that was present at Millennium when I joined there."

 

Miles has been in the biotech business for 25 years: in 1978, after receiving his PhD in molecular biology from University College in London, Miles joined Amersham as an R&D scientist. In 1992, he left Amersham to join Ribogene, a tiny instrument startup that turned into a drug discovery company, he said. He joined Millennium after a year at Dana Farber Cancer Institute as head of technology transfer.


 

Alnylam's rival RNAi therapeutics company, Sirna Therapeutics of Boulder, Colo.,  has also augmented its team, announcing July 29 that Scott Greer had joined its board of directors. Greer is currently chairman of the board at Abgenix. He was CEO of Abgenix from 1996 to 2002. Prior to that, he was senior vice president of corporate development and chief financial officer at Cell Genesys, and also served as director of corporate development at Genetics Institute. He also currently serves on the boards of directors at CV Therapeutics and Illumina.


            Amersham, which has not yet expressed much interest in RNAi, mentioned in its first half conference call July 29 that it has developed RNAi application protocols for its Oligo Pilot instrument, a benchtop oligo synthesis machine. "We see a little bit of uptake and a lot of interest in that," said Peter Ehrenheim,  the company's vice president of protein separations. "I don't think we see any significant numbers coming out of RNAi at this time. But in the future I am pretty sure that it will grow," he said.
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