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Strands: Dec 31, 2008

Mass. Life Sciences Center Awards $1.5M to Support Therapeutic RNAi Efforts
The Massachusetts Life Sciences Center announced earlier this month that it had awarded nearly $1.5 million to support two collaborations between industry and academia focused on developing RNAi delivery technologies.
According to the center, the money will be matched dollar-for-dollar by the industry partners involved in the partnerships.
The first grant is worth $250,000 a year for three years, and was awarded to Judy Lieberman of Harvard Medical School’s Immune Disease Institute and Baxter Healthcare subsidiary Epic Therapeutics to develop siRNA-containing microparticles as a treatment for human papillomavirus and herpes simplex virus II.
In October, RNAi News first reported the partnership between Lieberman and Baxter, which is focused on using Epic’s Promaxx microsphere technology for siRNA delivery (see RNAi News, 10/30/2008). 
The second grant, worth $249,593 per year for three years, was awarded to University of Massachusetts Medical School researchers Michael Czech and Gary Ostroff, along with RXi Pharmaceuticals, to fund their efforts to develop an oral delivery technology for RNAi drugs.
As reported by RNAi News in October, the technology, called glucan-encapsulated siRNA particles or GERPs, is based on the activity of M cells, which are specialized cells located in the small intestine (see RNAi News, 10/23/2008).
As part of the body’s defense mechanism, M cells are designed to take up antigens, such as bacteria or yeast, and transcytose them through the intestinal wall so they can be taken up by macrophages. Since their surface is made of beta-glucan, GERPs are taken up by M cells in the same manner as, for example, bacteria.
Under normal conditions, when a macrophage has taken up an antigen, it begins producing cytokines, which in turn triggers inflammation. But if treated with an siRNA, the GERP could be designed to inhibit inflammation at a specific site. 

Alnylam Files IND for Systemic Liver Cancer Drug Candidate
Alnylam Pharmaceuticals announced last week that it has submitted an investigational new drug application to US regulators for its systemic liver cancer drug candidate, ALN-VSP.
The drug includes two siRNAs — one targeting kinesin spindle protein and one targeting vascular endothelial growth factor — formulated in a lipid nanoparticle developed by partner Tekmira Pharmaceuticals.
Previously, Alnylam announced that in a mouse model, ALN-VSP triggered significant, dose-dependent silencing of its two targets. In addition, Alnylam researchers observed in treated mice histologically documented evidence of cell cycle arrest due to KSP silencing, a reduction in overall tumor growth as measured by quantification of a tumor-specific gene, and marked reduction in the size of liver tumors as observed by gross pathology.
“Following FDA review of our submission, we expect to initiate patient dosing in the first half of 2009, which positions us solidly on track to meet our goal of having three programs in clinical trials next year,” Akshay Vaishnaw, Alnylam’s senior vice president of clinical research, said in a statement.
According to Tekmira, it is eligible to receive an undisclosed milestone payment upon dosing of the first patient in the planned clinical study of ALN-VSP. The company said it could receive up to $16 million in milestones related to the drug’s development.

Abbott Clears US Antitrust Hurdle for Ibis Buy
Abbott said last week that it has received clearance from US antitrust regulators to proceed with the acquisition of Isis Pharmaceuticals’ molecular diagnostics subsidiary Ibis Biosciences.
The clearance comes less than a week after Abbott said that it had exercised its option to acquire the remaining 81.4 percent stake of Ibis that it didn’t already own for $175 million.
The acquisition is subject to other terms and conditions of a stock purchase agreement between the parties, but it is expected to close in January 2009.
Earlier this year, Abbott acquired an 18.6 percent stake in Ibis in two tranches for $40 million. At that time, it took an exclusive option to acquire the remaining interest in Ibis by June 30, 2009, for $175 million to $195 million, plus an earn-out tied to sales of instruments and assay kits.
Ibis sells the Ibis T5000 Biosensor System, which is used for rapid identification and characterization of infectious agents.

The Scan

Expanded Genetic Testing Uncovers Hereditary Cancer Risk in Significant Subset of Cancer Patients

In Genome Medicine, researchers found pathogenic or likely pathogenic hereditary cancer risk variants in close to 17 percent of the 17,523 patients profiled with expanded germline genetic testing.

Mitochondrial Replacement Therapy Embryos Appear Largely Normal in Single-Cell 'Omics Analyses

Embryos produced with spindle transfer-based mitochondrial replacement had delayed demethylation, but typical aneuploidy and transcriptome features in a PLOS Biology study.

Cancer Patients Report Quality of Life Benefits for Immune Checkpoint Inhibitors

Immune checkpoint inhibitor immunotherapy was linked in JAMA Network Open to enhanced quality of life compared to other treatment types in cancer patients.

Researchers Compare WGS, Exome Sequencing-Based Mendelian Disease Diagnosis

Investigators find a diagnostic edge for whole-genome sequencing, while highlighting the cost advantages and improving diagnostic rate of exome sequencing in EJHG.