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Alnylam Pools IP for ‘RNA Activation’ Tech From UT-Southwestern, UCSF, Salk Institute

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RNAi drug maker Alnylam Pharmaceuticals said this week that it has signed exclusive licensing deals with three academic institutions to consolidate intellectual property in the area of RNA activation.
 
Alnylam said that it has now gained a “dominant IP position” in the emerging field of RNA activation, or RNAa, after licensing a cache of intellectual property comprising mostly patent applications from three US research institutions.
 
Together, the agreements with the University of Texas Southwestern Medical Center; the University of California, San Francisco; and the Salk Institute for Biological Studies, also provide an example of how multiple entities can increase the chances of negotiating a deal for an emerging biotechnology by pooling relevant IP.
 
“It’s a collaborative effort,” Stuart Pollard, vice president of scientific and business strategy for Alnylam, told BTW this week. “They obviously agreed to come together, pool resources, and move this forward. The idea here is that it requires the sort of industrial muscle that a company like Alnylam has to move this toward therapeutic development. That takes work, and usually collaborative work. There is usually not one group that provides a solution in this regard.”
 
Alnylam, based in Cambridge, Mass., has traditionally played in the RNAi space, but the company is now committed to “scientific and IP leadership across all areas” of RNA, CEO John Maraganore said in a statement. This includes RNAi, microRNA, and now RNAa, which uses double-stranded modulatory RNA molecules to upregulate genes.
 
“Many human diseases are caused by the abnormally low expression of proteins, and RNAa could be used to treat these disorders through selective transcriptional gene activation,” Maraganore said.
 
Pollard added that RNAa is distinct from, but in some respects complementary to, the company’s RNAi drug development.
 
“RNAa is using similar molecules but leveraging a quite different mechanism, and offers a complete new platform, potentially,” Pollard said. “It’s a slightly earlier stage, but here you have the ability to switch on genes selectively, or at least that’s the vision we have for this. So it’s complementary, but represents a quite distinct and alternative platform using RNAi molecules.”
 
According to Pollard, the phenomenon behind RNAa has been known for several years, but the mechanism behind it was slow to be revealed. That all changed in recent years with the publication of several peer-reviewed papers describing work done in the laboratories of David Corey, a professor of pharmacology and biochemistry at UT Southwestern; Fred Gage, a professor and chair for research on age-related neurodegenerative diseases at the Salk Institute; and Long-cheng Li, an assistant professor of urology at UCSF.
 
“There has been some degree of controversy about this area, but there has been some real progress that has led, in part, to the development of this IP,” Pollard said.
 
Specifically, Alnylam said that the papers have provided evidence that RNAa is mediated by double-stranded RNAs, possibly including endogenous miRNAs that target the promoter regions of genes in chromosomal DNA.
 
“So-called ‘anti-gene RNAs,’ or agRNAs, appear to increase transcription by complementary base-pairing with corresponding promoter sequences which enhance recruitment of the cellular RNA polymerase II and therefore increase the target gene’s expression,” Alnylam said.
 
The company also said that in vitro studies have demonstrated that agRNAs can increase the levels of a target gene’s mRNA by nearly tenfold, which increases the production of that gene’s protein.
 
Alnylam scientists and executives have maintained relationships with researchers in the labs at the three institutions over the years, which is in general how it became aware of the breakthrough work in each laboratory, Pollard said.
 
In addition, the company’s strategy of being proactive in publishing its results in peer-reviewed journals has made researchers in the field aware of Alnylam’s activities, and as such the company is often approached by researchers with emerging RNA-related technologies, he added.
 
Although the company did not previously have sponsored research programs in place with any of the institutions prior to the licensing agreements, it now plans to forge official research collaborations with the groups to move the RNAa in to therapeutic applications, Pollard said.
 
“We felt, as a group that wants to play a lead role in this space, it was time to pull that IP into our estate, and also to collaborate with these folks and develop this science,” Pollard added. “I think they want to see their technologies developed and … [that] there is a mutual respect and desire to work together.”

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