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Alnylam, Rosetta Genomics, US Genomics, Lentigen, UPenn, Qiagen, CytoPathfinder, CytRx, Bioneer, Sigma-Aldrich, Rosetta, Sirna

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Alnylam Provides Updates on Respiratory Programs at ATS Meeting

Alnylam Pharmaceuticals reported this week preclinical data from its pandemic flu, asthma, chronic obstructive pulmonary disorder, and idiopathic pulmonary fibrosis drug-development programs.

The company also said that it now anticipates its flu therapy will feature two siRNAs targeting different genes within the flu virus genome in order to create broad antiviral activity while reducing the likelihood of viral resistance. Alnylam expects to file an investigational new drug application for a flu drug, which is being developed with Novartis, by the end of the year.

"We and our collaborators are committed to exploring novel therapeutics for the treatment of many respiratory diseases," Antonin de Fougerolles, senior director of research at Alnylam, said in a statement. "The preclinical findings we announced … represent continued progress with our pandemic flu program, as we advance this effort toward the clinic."

The preclinical data were presented at the American Thoracic Society International Congress in San Diego this week, Alnylam said.

According to Alnylam, it has demonstrated in vitro antiviral activity toward a human clinical isolate of the avian flu virus, also known as H5N1. "These potent effects toward H5N1 were achieved with multiple siRNAs that also showed antiviral activity toward other flu strains," the company said.

Alnylam noted that its flu drug will target sequences common to all flu genomes, not just H5N1.

Alnylam said that at the ATS meeting it presented data showing that respiratory delivery of siRNAs targeting IKK2, a gene associated with inflammatory responses that is also known as IKK-beta, was able to inhibit expression of the gene in the lung of an animal model.

"In addition, results showed robust silencing of the expression of pro-inflammatory cytokines such as TNF-alpha and IL-1beta," the company said. The work was done in collaboration with researchers from Imperial College.

"In vitro optimization of siRNAs targeting IKK2 was achieved, and siRNAs were generated that demonstrated robust and specific inhibition of IKK2 mRNA and protein, with no effects on a related family member, IKK1 — also known as IKK-alpha," according to Alnylam. "These results offer the possibility of designing an RNAi therapeutic that is able to both selectively and locally inhibit IKK2 activity in lung for the treatment of respiratory diseases, such as asthma or COPD."

Alnylam also provided an update of a program exploring local delivery of siRNAs to treat IPF, a potentially fatal pulmonary fibrotic disorder of unknown origin. Through a collaboration with the University of Giessen Lung Center in Germany, Alnylam said it has identified and is evaluating in animal models siRNAs targeting multiple members of the TGF-beta pathway.


Rosetta Genomics, US Genomics to Collaborate on Lung Cancer Dx

Rosetta Genomics said this week that it will collaborate with US Genomics on the development of a microRNA-based, non-invasive diagnostic for lung cancer.

Under the terms of the deal, the companies will combine US Genomics' Trilogy 2020 platform and Direct miRNA assay with Rosetta's proprietary miRNAs. Using the miRNAs as biomarkers, the companies expect to be able to identify lung cancer using a sputum or blood sample.

Additional terms of the deal were not disclosed.


Lentigen Licenses UPenn's Lentiviral Vector Tech for Reagent, Dx Development

Lentigen said this week that it has received an exclusive worldwide license from the University of Pennsylvania to use its lentiviral vector technology.

According to the company, the technology can be used to deliver genes or RNAi molecules into cells. The license gives Lentigen the right to use the technology to develop research reagents and therapeutics.

Financial terms of the arrangement were not disclosed.


Qiagen, CytoPathfinder to Develop RNAi Screening Tools

Qiagen and CytoPathfinder said this week that that have formed a strategic collaboration to develop tools for high-throughput RNAi screening.

The arrangement would combine Qiagen's siRNA sets with CytoPathfinder's transfection microarray technology, which allows for siRNA screening on a microarray chip.

Additional terms of the deal were not disclosed.


CytRx Posts Higher Q1 Net Loss on Increased Expenses, Continues to Plan RNAi Drug Spin Out

CytRx reported this week its first-quarter financial results, posting an increased net loss amid sharply higher general and administrative costs.

The company also said that it still intends to spin out its RNAi drugs operations, but that it has not secured the funds to do so.

CytRx's net loss in the quarter rose to $4.2 million, or $0.07 per share, from $3.5 million, or $0.07 per share, in the year-ago quarter.

Research and development spending was down slightly, to $1.7 million from $1.9 million, while general and administrative costs climbed to $2.6 million from $1.7 million. The higher general and administrative expenses in part reflect increased salary expense associated with the hiring of additional employees, the company said.

Revenues in the first quarter, which consisted entirely of service revenue, were $61,000, up from $1,500 in the same period last year.

As of March 31, CytRx had cash, cash equivalents, and short-term investments totaling $17.1 million.

CytRx said that formation of its RNAi drugs subsidiary is expected to occur "in the coming months, subject to obtaining financing." The company first disclosed its intent to spin out the business unit in an interview with RNAi News in November last year (see RNAi News, 11/11/2005).


Alnylam Grants Bioneer License to RNAi Patents for Research Products

Alnylam Pharmaceuticals announced this week that it has granted South Korean oligo maker Bioneer a non-exclusive license to use its Kreutzer-Limmer patent family for research product development and commercialization.

The patents relate to the use of siRNA in mammalian cells.

"This agreement brings the number of licenses Alnylam has executed granting rights to its intellectual property estate to more than 20, including 12 with research product suppliers," Roland Kreutzer, managing director of Alnylam Europe, said in a statement. "In addition, we believe that a significant portion of sales of siRNAs for research purposes are currently being made under access to Alnylam's intellectual property."

Terms of the licensing deal were not disclosed.


Sigma-Aldrich Licenses Rosetta Inpharmatics' siRNA Informatics Technology

Sigma-Aldrich this week said has acquired an exclusive license to Rosetta Inpharmatics' bioinformatics design tools for siRNA research and development purposes.

Sigma-Aldrich said it plans to use Rosetta' design technology to launch human and model organism siRNA whole-genome libraries, to deliver siRNA panels targeted to specific gene families, and to provide access to single-target pre-designed siRNAs through its website.

Rosetta is a wholly owned subsidiary of Merck.

Financial details were not disclosed.


Sirna Prices Stock Offering at $5 Per Share

Sirna Therapeutics this week priced its offering of 9 million common shares at $5 per share.

The offering, which will gross the company about $45 million, was increased from its previously announced size of 8 million shares, the company noted.

The Scan

US Booster Eligibility Decision

The US CDC director recommends that people at high risk of developing COVID-19 due to their jobs also be eligible for COVID-19 boosters, in addition to those 65 years old and older or with underlying medical conditions.

Arizona Bill Before Judge

The Arizona Daily Star reports that a judge weighing whether a new Arizona law restricting abortion due to genetic conditions is a ban or a restriction.

Additional Genes

Wales is rolling out new genetic testing service for cancer patients, according to BBC News.

Science Papers Examine State of Human Genomic Research, Single-Cell Protein Quantification

In Science this week: a number of editorials and policy reports discuss advances in human genomic research, and more.