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Icoria, TGen in New NIEHS RNAi Initiative


The National Institute of Environmental Health Sciences launched a three-year project to develop a library of siRNA sequences targeting a number of human genes believed to be associated with environmentally related diseases.

The project, being conducted by the Translational Genomics Research Institute with the help of Icoria, is the first phase of a broader effort to develop an RNAi resource for the members of the NIEHS’s National Center for Toxicogenomics.

According to Ray Tennant, director of the NCT and head of the NIEHS’s RNAi initiative, the center’s microarray group collaborates with scientists from five institutions with the goal of bringing “definition to the field of toxicogenomics, which we view as the interface between science and [the] adverse effects of chemicals, … drugs, … and environmental influences.”

The groups, which work with each other as the Toxicogenomics Research Consortium, aim to identify biomarkers that would allow researchers to predict adverse effects, rather than simply respond to them once they happen, Tennant says.

Over its roughly three-year life, the TRC has relied heavily on gene-expression data gathered through microarray analysis, Tennant says.

“That’s where the RNAi project comes into play,” he says. “It is one of the functional [technologies] we are trying to use to make a link between the data obtained from microarray analysis and potential causal relationships to adverse phenotypes that may be induced by chemical or environmental stressors. [The RNAi initiative’s goal] is to give [us] … another tool in the toolbox to take the science forward.”

— Doug Macron



US Patent application 20040248839. RNAi targeting of viruses. Inventor: Timothy Kowalik. Assignee: University of Massachusetts. Filed: February 5, 2004.

According to the abstract, this invention covers methods to inhibit replication of viruses, such as a cytomegalovirus, in a host or host cell. “Methods and compositions of the invention utilize RNA interference to block the translation of mRNA into proteins which are important or essential to viral replication. The method and compositions can be used to study CMV infection in in vitro cell culture and to treat CMV infection in non-human primates and human subjects,” the abstract says.


US Patent application 20040229266. RNA interference mediating small RNA molecules. Inventors: Thomas Tuschl, Sayda Mahgoub Elbashir, Winfried Lendeckel. Assignee: Max-Planck-Gesellschaft zur Forderung Der Wissenschaften. Filed: April 27, 2004.

Working in Drosophila, the inventors use 19-23 nt short RNA fragments to prove they work as “sequence-specific mediators of RNAi,” the abstract states. “Chemically synthesized siRNA duplexes with overhanging 3’ ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.”






The ID number of Benitec’s core patent in Australia which is at issue in the ongoing suit between that company and Nucleonics. Benitec originally sued several companies, including Nucleonics, for patent infringement; the other companies settled, but Nucleonics instead challenged the validity of Benitec’s patents. The Australian patent office has reviewed two related patents and has begun proceedings that could result in the patents’ being overturned.


Acuity Pharmaceuticals announced late last year that its siRNA-based treatment for age-related macular degeneration has been given to patients in a phase I trial, marking the first time an RNAi therapeutic has been used in humans.


Isis Pharmaceuticals said that the first antisense drug to emerge from its work with Eli Lilly has moved into phase I testing. The drug targets a molecule called survivin, which is found in many types of tumors. Isis will receive a $1.5 million milestone payment for this step.


Alnylam Pharmaceuticals reported a net loss of $6.4 million for the third quarter of last year, compared to a loss of $12 million in the same quarter the year before. R&D spending was up slightly to $4.8 million, and overall expenses fell by more than $3 million to $7.8 million. Revenues, which had been $74,000 in the quarter in 2003, rose to $1.4 million in 2004.


Scientists at Virginia Polytechnic Institute have started a project to build a computational model of the RNAi process as it occurs in C. elegans.


Sirna Therapeutics announced its first RNAi-based program related to dermatology with an initial focus on hair removal. Sirna Dermatology plans to begin human trials next year. In other Sirna news, the company late last year was expected to acquire a private company, based on information it filed with the SEC in November.



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