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Mirus Debuts ‘New’ Veteran Gene Function Tool


In late September, Mirus Bio announced the availability of its hydrodynamic delivery technology through licensing and service deals for RNAi research applications.

While the company billed the delivery approach as a “new tool” for gene function analysis, the technique has actually been available for years. According to David Lewis, RNAi program director at Mirus, the decision to call the technology new stemmed from a lack of awareness among researchers.

“Hydrodynamic [delivery] through the tail vein has been around since 1999, [and for siRNAs, the approach] was first demonstrated in 2002,” he says. “But, when we talk to people, it’s amazing how many people haven’t heard about hydrodynamic injection or what it’s for or what it allows you to do.

“So … we called it new just because we realized there is a big audience of people who are thinking about using RNAi in vivo but may not be familiar with [the] technology,” he says.

Richard Schifreen, vice president of research products at Mirus, added that the time seemed right to launch a concerted effort to commercialize the technology after the company received the go-ahead from partner Rosetta Inpharmatics to publish data from work the companies did comparing the hydrodynamic delivery method with traditional gene knockout.

“For some time, we’ve had a number of different collaborations and service-type work we’ve done for people … [but] with a lot of the work we’ve done and continue to do, our partner or customer wants to keep the work proprietary,” he says.

But with Rosetta open to publishing the data, “the demonstration is now out there for the public … [and] we’re ramping up in terms of our commercialization,” Schifreen adds. Mirus is “trying to do more to get the word out that this will work, exactly what it can do, and our willingness to work with people in a flexible way to allow them to use this technology.”

— Doug Macron



US Patent 7,112,405. Compositions and Methods for Enhancing Oligonucleotide-Mediated Gene Alteration. Inventors: Eric Kmiec, Michael Rice, and Li Liu. Assignee: University of Delaware. Issued: September 26, 2006. According to the patent’s abstract, the invention comprises “compositions and methods for enhancing oligonucleotide-directed nucleic acid sequence alteration in vivo, ex vivo, and in vitro. … These methods and compositions involve cells and cell-free extracts with altered levels or activities of a protein from the RAD52 epistasis group, the mismatch repair group, and/or the excision repair group.”

US patent application 20060212950. Inducible Small RNA Expression Constructs for Targeted Gene Silencing. Inventors: Thomas Tuschl, Tilmann Achsel, Reinhard Luhrmann, and Jutta Meyer. Assignee: Max Planck Institute. Filed: January 15, 2004. This invention relates to a recombinant vector for the inducible expression of a single-stranded or double-stranded RNA molecule in a eukaryotic, particularly mammalian, cell comprising at least one sequence coding for the RNA molecule operatively linked to an expression control sequence comprising a polymerase III promoter and at least one regulatory protein binding site and optionally a transcription terminator.



Quark Biotech has landed the world’s biggest pharmaceutical firm as a partner by exclusively licensing a proprietary human gene target to Pfizer for certain indications, including age-related macular degeneration. Under the deal, Pfizer acquired RTP-801i, an siRNA-based molecule that silences RTP-801 and which Quark expects to move into phase I testing before the end of the year.  

Luminex has signed a deal with Exiqon to develop products combining key technologies for miRNA detection. The products will marry Luminex’s xMAP bioassay technology with Exiqon’s Locked Nucleic Acids and will be the first miRNA offerings from Luminex.  

Cenix BioScience has joined forces with Merck to provide services to boost the German pharma’s drug target discovery and validation efforts in oncology. Under the agreement, Cenix will apply its expertise combining genome-scale screening applications of RNAi with high-content phenotypic cell analyses to discover and validate new therapeutic targets, using assays co-designed with Merck scientists.  

According to researchers at IBM and the Genome Institute of Singapore, current estimates of the number of microRNA genes and targets in the human genome may be off by an order of magnitude or more. Publishing in Cell, the team suggests that the number of miRNAs could number in the tens of thousands, while some of these may have a few thousand targets.



$1.9 million

Amount Nastech Pharmaceutical received from the US National Institute of Allergy and Infectious Diseases to support the company’s development of an RNAi-based influenza therapy.

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