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Sirna, Open Biosystems, Ambion

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Sirna Selects HCV Drug Candidate

Sirna Therapeutics said this week that it has selected an RNAi compound for development as a hepatitis C therapeutic.

According to the company, the compound, called Sirna-AV34, is a systemically delivered, nanoparticle-based, chemically modified short interfering RNA that targets multiple sequences of the HCV genome. It is designed to inhibit viral replication and reduce the selection of drug-resistant mutant variants.

Sirna said that it has begun cGMP manufacturing of the compound for phase I clinical studies. Investigational new drug application-enabling toxicology studies are slated to start in the first quarter of 2006, with an IND filing expected in the fourth quarter.


Open Biosystems Signs Deals for RNAi Tech with Cancer Research Institutes

Open Biosystems said this week that it has signed on Duke University, the National Institutes of Health's National Cancer Institute, Lee Moffitt Cancer Center, and Fox Chase Cancer Center as customers for its short hairpin RNA libraries for the identification of cancer treatment options.

According to Open Biosystems, the RNAi libraries can be used to screen entire genomes in a high-throughput manner, identifying tumor suppressors and possible drug targets.

"Open Biosystems' shRNAmir libraries enable us to manipulate gene expression and probe gene function on a whole-genome scale, accelerating a wide range of basic and translational research programs, including cancer research," Thomas Burke, general manager of technologies at the Duke Institute for Genome Sciences & Policy, said in a statement. "Rather than spending limited time and resources designing, constructing, and characterizing RNAi reagents, our researchers can now select shRNAmir clones targeting genes of interest, and place these into functional assays accelerating research that may lead to advanced treatment options for cancer."

Specific terms of the deals were not disclosed.


Ambion Delivers siRNA Libraries to European Research Groups

Ambion said this week that it has made the final shipment of its genome-wide human siRNA library to the Max Planck Institute for Molecular Cell Biology and Genetics.

The company also said that it has completed delivery of a Silencer genome-wide human siRNA library to Europe's MitoCheck consortium.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.