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IP Update: Recent Patents, Patent Applications Awarded to Isis; Allergan; Stanford; and More

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Title: Transcriptional Regulation of Gene Expression by Small Double-Stranded Modulatory RNA

Patent Number: 8,092,992

Filed: May 28, 2004

Lead Inventor: Tomoko Kuwabara, Salk Institute

“The invention provides a method for modulating gene expression by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes, wherein the association results in altered expression of the one or more genes,” the patent's abstract states. “The invention is further directed to a method for directing the differentiation of neuronal stem cells into neurons by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes involved in neuronal differentiation and directing the transcription of the one or more genes. In related embodiments, the invention provides particular compositions of double-stranded ribonucleic acid molecules, as well as therapeutic and screening applications of the invention.”


Title: Methods of Determining Potency of Chemically Synthesized Oligonucleotides

Patent Number: 8,093,061

Filed: Oct. 18, 2007

Lead Inventor: Walter Lima, Isis Pharmaceuticals

The patent, its abstract states, claims “methods for determining potency of RNAi agents. Such methods include, but are not limited to, cell-based and cell-free assays that measure binding of an RNAi agent with Ago2 or that measure Ago2 activity in the presence of such RNAi agents. Also provided are assays that determine potency of RNAi agents by assessing their ability to compete with other RNAi agents, including control RNAi agents, for binding and/or activation of Ago2.”


Title: RIP140 Regulation of Diabetes

Patent Number: 8,093,223

Filed: Oct. 8, 2009

Lead Inventor: Michael Czech, University of Massachusetts (RXi Pharmaceuticals)

“Inhibition of RIP140 increases glucose transport,” the patent's abstract states. “Compounds that inhibit RIP140 expression or activity are useful for treating disorders associated with aberrant glucose transport, treating obesity, increasing metabolism, and increasing brown fat.”


Title: Compositions for Silencing the Expression of VDAC1 and Uses Thereof

Patent Number: 8,093,369

Filed: Oct. 15, 2006

Lead Inventor: Varda Shoshan-Barmatz, Ben Gurion University

The invention, the patent's abstract states, “relates generally to the down-regulation of mitochondrial protein, voltage-dependent anion channel, expression by RNAi, or antisense therapy. In particular, the ... invention is directed to VDAC1 silencing molecules useful in regulating cell proliferation and to pharmaceutical compositions comprising same useful in the treatment of diseases associated with aberrant cell proliferation.”


Title: siRNA Targeting Spleen Tyrosine Kinase

Patent Number: 8,093,370

Filed: Dec. 6, 2010

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.”


Title: Compositions and Methods for the Treatment of Ophthalmic Disease

Application Number: 20120003275

Filed: June 10, 2011

Lead Inventor: Gerard Rodrigues, Allergan

The invention comprises “compositions and methods of treating ocular disorders [using] CXCR4 inhibitory compositions,” according to the patent application's abstract.


Title: Single-Walled Carbon Nanotubes/siRNA Complexes and Methods Related Thereto

Application Number: 20120003278

Filed: July 1, 2011

Lead Inventor: Lynn Kirkpatrick, Ensysce Biosciences

The invention “includes single-walled carbon nanotube compositions for the delivery of bioactive agents and methods of making such single-walled carbon nanotube compositions,” the patent application's abstract states.


Title: Targeted Delivery System

Application Number: 20120004181

Filed: July 22, 2011

Inventor: Lali Medina-Kauwe, Cedars-Sinai Medical Center

The invention, the patent application's abstract states, comprises a “targeted delivery system for siRNA or antisense technology. In one embodiment, the invention provides for a method of treating cancer by administering a therapeutically effective dosage of HerPBK10 combined with siRNA, resulting in the inhibition of Her2 expression and cell death. In another embodiment, a plurality of HerPBK10 combined with siRNA form a nanoparticle.”


Title: lincRNAs in Cancer Diagnosis and Treatment

Application Number: 20120004278

Filed: June 17, 2011

Lead Inventor: Howard Chang, Stanford University

“Long non-coding RNAs, a relatively recently recognized class of widely transcribed genes, are thought to affect chromatin state and epigenetic regulation, but their mechanisms of action and potential roles in human disease are poorly understood,” the patent application's abstract states. The invention “shows that long non-coding RNAs in the human HOX loci are systematically dysregulated during breast cancer progression, and that expression levels of the lincRNA termed HOTAIR can predict cancer metastasis. Elevated levels of HOTAIR can lead to altered patterns of Polycomb binding to the genome. These findings indicate that lincRNAs have active roles in modulating the cancer epigenome and may be important targets for cancer diagnosis and therapy.”


Title: RNA Interference-Mediated Inhibition of the High Affinity 1 gE Receptor Alpha Chain Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20120004280

Filed: March 25, 2010

Lead Inventor: Vasant Jadhav, Merck

The invention, the patent application's abstract states, “relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of FC-epsilon-R1-alpha gene expression and/or or activity, and/or modulate a FC-epsilon-R1-alpha gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules ... capable of mediating or that mediate RNA interference against FC-epsilon-R1-alpha gene expression.”


Title: RNA Interference-Mediated Inhibition of the Nerve Growth Factor Beta Chain Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20120004281

Filed: March 25, 2010

Lead Inventor: Vasant Jadhav, Merck

The invention relates to “compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of NGF-beta gene expression and/or activity, and/or modulate a NGF-beta gene expression pathway,” according to the patent application's abstract. “Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules ... capable of mediating or that mediate RNA interference against NGF-beta gene expression.”


Title: RNA Interference-Mediated Inhibition of the Intercellular Adhesion Molecule 1 Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20120004282

Filed: March 25, 2010

Lead Inventor: Victoria Pickering, Merck

The invention relates to “compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of ICAM-1 gene expression and/or activity, and/or modulate an ICAM-1 gene expression pathway,” the patent application's abstract states. “Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules … capable of mediating or that mediate RNA interference against ICAM-1 gene expression.”


Title: Novel shRNA Gene Therapy for Treatment of Ischemic Heart Disease

Application Number: 20120004283

Filed: Sept. 15, 2009

Lead Inventor: Joseph Wu, Stanford University

“Short hairpin RNA interference therapy targeting hypoxia inducible factor prolyl-4-hydroxylase protein is used for treatment of myocardial ischemia,” the patent application's abstract states. “This treatment can be followed non-invasively by molecular imaging. Provided are compositions comprising novel vectors encoding shRNA targeting the HIF-1-alpha and asparaginyl hydroxylase genes. The vectors encoding shRNA are also useful for the treatment of cardiac diseases, peripheral vascular diseases, and decubitis ulcers.”


Title: RNA Interference-Mediated Inhibition of TNF and TNF Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20120004403

Filed: June 21, 2011

Lead Inventor: Leonid Beigelman, Merck

“This invention relates to compounds, compositions, and methods useful for modulating tumor necrosis factor and/or tumor necrosis factor receptor gene expression using short interfering nucleic acid molecules,” the patent application's abstract states. “This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of tumor necrosis factor and/or tumor necrosis factor receptor gene expression and/or activity by RNA interference using small nucleic acid molecules.”

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