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Follow the leader
CAMBRIDGE, Mass.—Alnylam Pharmaceuticals Inc. in early August announced that it had consolidated the key intellectual property (IP) for RNA activation (RNAa), creating what the company is calling "a dominant IP position in this new area of biology." To consolidate IP in this field, Alnylam recently completed exclusive license agreements with the leading academic institutions working on RNAa: the Corey lab at the University of Texas Southwestern Medical Center; the Li lab at the University of California San Francisco; and the Gage lab at the Salk Institute for Biological Studies.
"RNA activation represents potentially a new way of controlling gene expression in cells, which, in turn, could create an entirely new platform for developing novel therapeutics targeting a range of disease areas, including certain genetic diseases and cancer where the aberrant low expression of certain proteins is known to occur," says Dr. John Maraganore, CEO of Alnylam.
However, when asked what areas of discovery and development are most likely to be impacted in the short run by RNAa advances, he notes that it is "premature at this time to provide guidance on timelines for specific programs."
RNAa is an emerging biological discovery involving double-stranded RNAs that target promoter regions in chromosomal DNA, resulting in transcriptional activation of genes. The transcriptional activation, or up-regulation, of genes results in an increase in mRNA and protein production. It is an area of research that Alnylam couldn't ignore given its ongoing efforts to be a leader in RNA interference (RNAi) and microRNAs, Maraganore notes.
"Alnylam is committed to scientific and IP leadership across all areas of modern biology's RNA revolution," Maraganore says. "As such, we have been following the development of RNA activation, as well as working with the key leaders in this space for some time now, and felt that it had reached a point of maturity and scientific validation where we should start consolidating the IP."
Obtaining exclusive access to these key RNAa patents puts Alnylam in a position "to lead in the translational research of this biology as it advances toward in vivo validation and provides a solid foundation for business execution," he says, "as we've demonstrated in the past with our RNAi and microRNA platforms."
It's too early in development to say what relation RNAa might have to Alnylam's RNAi and microRNA therapeutics work and where it might interact with or bolster efforts in those other areas, Maraganore notes, "but we believe it is important to understand all aspects of the biology around RNA," he says.
"We're excited to be working with Alnylam, as they have demonstrated a commitment to scientific excellence in their publications and academic collaborations," said Dr. David Corey, a professor of pharmacology and biochemistry at UT Southwestern, in a news release about the IP consolidation. "We look forward to collaborating on the emerging science of RNAa biology to further explore the breadth of applications for transcriptional gene activation with anti-gene RNAs."
Alnylam is continuing to work with the key leaders in the field of RNA activation to better understand the breadth of this new area of biology, Maraganore says, and although there are no specific immediate plans to acquire or license addition RNAa IP, he adds that "we will continue to consolidate IP as we see necessary to maintain a dominant IP position." DDN