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Altogen Looks Toward Therapeutic siRNA Delivery


Looking to extend the use of its research products into the therapeutics field, Altogen Biosystems has begun exploring whether its transfection technologies can be used to deliver siRNA-based drugs.

When it comes to research, "each of our customers has their own focus for delivery, [whether it] be siRNAs, microRNAs, [or] antibodies, and we offer the tools," says Andreas Kim, vice president of research and development at Altogen.

But recognizing the growing interest in the therapeutic applications of RNAi, the company has also started testing the waters of drug delivery, with a particular focus on siRNAs, he adds.

To take advantage of that opportunity, Altogen has launched a research effort to use its technologies to address the "number of variables" associated with drug delivery, most notably protection of a therapeutic payload and avoidance of toxicity and immune responses, he says.

More specifically, Altogen has been working on how to best design its delivery vehicles so that they are naturally delivered to target tissues and organs, Kim notes. In conjunction with undisclosed collaborators, the company is also investigating the use of targeting molecules such as aptamers and antibodies in order to deliver
siRNAs to specific parts of the body.

Thus far, Altogen has primarily been working on delivery to tumors and the liver, Kim says. But the company is also investigating delivery to the brain using its reagents that are able to cross the blood-brain barrier.

He cautions, however, that "there are still studies that need to be done to make sure that [the delivery vehicle] is non-toxic [and] that it actually can cross the blood-brain barrier not only by itself, but when it is conjugated to a drug."
Even though Altogen's drug delivery program is early stage, Kim says that the company already has generated a limited amount of siRNA data that it has been showing to potential pharmaceutical partners.

— Doug Macron

RNAi Notes

Roche will file its first investigational new drug application for an siRNA therapeutic in 2010. That agent will be delivered using Tekmira Pharmaceuticals' stable nucleic acid lipid particles technology.

US Patent and Trademark Office is issuing a patent that covers Kylin Therapeutics' core packaging RNA technology. Its pRNA is derived from bacteriophage phi29 and the company says it can be modified with therapeutic RNA such as siRNA.

Benitec will collaborate with the Children's Cancer Institute Australia for Medical Research to develop an RNAi-based therapeutic for lung cancer.

RXi Pharmaceuticals acquired direct ownership of a self-delivering RNAi technology that it had previously licensed from Advirna.


$16 million
Amount Opko Health will pay to acquire pharmaceutical firm Pharma Genexx.

Funded grants

$337,544/FY 2009
Investigate Stability of RNA Interference in Human Lymphocytes
Grantee: Dong Sung An, University of California, Los Angeles
Began: Aug. 1, 2009; Ends Ju1. 31, 2011

An and his colleagues will be studying whether siRNA has unintended, nonspecific toxic effect on human lymphocytes. They hypothesize that the higher the shRNA levels, the more likely a cytotoxic effect is. They will test that theory and optimize shRNA levels so that they are effective and not cytotoxic.

$353,846/FY 2009
Role of MicroRNAs in Mammalian Ear Development and Neurosensory Specification
Grantee: Garrett Soukup, Creighton University
Began: Jun. 2, 2009; Ends: May 31, 2011

Soukup's lab will study the role of miRNAs in the development of the mouse inner ear. They plan to study microRNA expression patterns related to ear biology, determine whether miRNAs are needed for neurosensory epithelial development and maintenance, and determine the function of miRNA in hair cell development and maintenance.

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