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IP Update: Jun 2, 2011


Title: RNA Interference Agents for Therapeutic Use

Patent Number: 7,951,784

Filed: Jan. 26, 2007

Lead Inventor: Tariq Rana, University of Massachusetts (RXi Pharmaceuticals)

“The invention features chemically modified small interfering RNAs that are stable in vivo and retain the ability to form an A-form helix when in association with a target RNA,” the patent's abstract states. “The featured siRNA are effective therapeutics, particularly for targeting SOD1.”

Title: siRNA Targeting V-Myc Myelocytomatosis Viral Gene Oncogene Homolog

Patent Number: 7,951,935

Filed: June 16, 2009

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to Myc.”

Title: Dock-and-Lock Complexes for Delivery of Interference RNA

Application Number: 20110123436

Filed: Dec. 9, 2010

Lead Inventor: Chien-Hsing Chang, IBC Pharmaceuticals

The invention, the patent application's abstract states, comprises “compositions and methods of use of targeted delivery complexes for delivery of siRNA to a disease-associated cell, tissue, or pathogen. The targeted delivery complex comprises a targeting molecule, such as an antibody or fragment thereof, conjugated to one or more siRNA carriers. In preferred embodiments, the siRNA carrier is a dendrimer or protamine and the targeting molecule is an anti-cancer antibody, such as hRS7.

“More preferably, the antibody or fragment is rapidly internalized into the target cell to facilitate uptake of the siRNA … [and] the targeted delivery complex is made by the DNL technique,” the abstract adds. “The compositions and methods are of use to treat a variety of disease states, such as cancer, autoimmune disease, immune dysfunction, cardiac disease, neurologic disease, inflammatory disease, or infectious disease.”

Title: Site-Specific Delivery of Nucleic Acids by Combining Targeting Ligands with Endosomolytic Components

Application Number: 20110123520

Filed: April 10, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for site-specific delivery of nucleic acids by combining them with targeting ligands and endosomolytic components,” the patent application's abstract states.

Title: IKK-Alpha- and IKK-Beta-Specific Inhibitors

Application Number: 20110124099

Filed: Aug. 3, 2010

Lead Inventor: Lun Li, Boehringer Ingelheim

The patent application, its abstract states, claims “a method for modulating NF-kappa-B-dependent gene transcription in a cell comprised of modulating IKK-alpha and IKK-beta protein and protein activity in the cell. The ... invention also provides siRNA compositions and methods thereof for modulating NF-kappa-B dependent gene transcription.”

Title: Use of miR-26 Family as a Predictive Marker for Hepatocellular Carcinoma and Responsiveness to Therapy

Application Number: 20110124521

Filed: June 11, 2009

Lead Inventor: Xin Wang, Fudan University (Ohio State University)

“Expression of microRNA-26 is decreased in hepatocellular tumor tissue relative to non-cancerous tissue, and ... a low level of microRNA-26 is associated with a poor clinical outcome,” the patent application's abstract states. Additionally, “low expression level of microRNA-26 is correlated with a favorable response to interferon-alpha therapy in HCC patients.”

The invention comprises “a method of predicting the clinical outcome of a patient diagnosed with HCC [by] detecting the level of microRNA-26 expression in a sample obtained from the patient,” the abstract states. “Also provided is a method of selecting a patient diagnosed with HCC as a candidate for IFN-alpha therapy, comprising detecting the level of microRNA-26 expression in a sample obtained from the patient. A method of identifying therapeutic agents for the treatment of HCC, comprising screening candidate agents in vitro to select an agent that increases expression of microRNA-26 in HCC cells [is] also provided … [as are] methods of treating a patient diagnosed with HCC and expressing a low level of miR-26 … [with] IFN-alpha therapy.”

Title: RNAi-Mediated Inhibition of Ocular Hypertension Targets

Application Number: 20110124708

Filed: Dec. 14, 2010

Lead Inventor: Allan Shepard, Alcon

The patent application, its abstract states, claims the use of “RNA interference ... for inhibition of ocular hypertension target mRNA expression for lowering elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension.”

Title: Composition and Methods of RNAi Therapeutics for Treatment of Cancer and Other Neovascularization Diseases

Application Number: 20110124710

Filed: Jan. 13, 2011

Lead Inventor: Yijia Liu, Intradigm (Silence Therapeutics)

The invention, the patent application's abstract states, comprises “compositions and methods ... for treatment of diseases involving unwanted neovascularization. The invention provides treatments that control NV through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological NV tissues. Tissue-targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference are provided.

“The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF-pathway antagonists,” the abstract adds. “The compositions and methods can be used for the treatment of NV diseases such as cancer, ocular disease, arthritis, and inflammatory diseases.”

Title: Compositions and Methods for Inhibiting Expression of Nav1.8 Gene

Application Number: 20110124711

Filed: Jan. 28, 2011

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the Nav1.8 gene, comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene,” the patent application's abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene in a cell.”

Title: Anti-Cancer Composition Comprising microRNA Molecules

Application Number: 20110124712

Filed: July 25, 2008

Lead Inventor: Jong Bae Park, National Cancer Center

The invention comprises an “anti-cancer composition for the treatment of hypoxia-induced angiogenesis-associated diseases including cancers,” the patent application's abstract states. “It comprises a microRNA-125 nucleic acid molecule. Also, methods of inhibiting angiogenesis, suppressing the invasion and metastasis of cancer cells, and treating cancers are provided.”

Title: Conjugates and Compositions for Cellular Delivery

Application Number: 20110124853

Filed: Sept. 27, 2010

Lead Inventor: Tongqian Chen, Merck

“This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide, and human serum albumin-derived conjugates of biologically active compounds, including … antisense, dsRNA, siNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys, and aptamers,” the patent application's abstract states.

The Scan

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Finding Safe Harbor in the Human Genome

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New Data Point to Nuanced Relationship Between Major Depression, Bipolar Disorder

Lund University researchers in JAMA Psychiatry uncover overlapping genetic liabilities for major depression and bipolar disorder.