Title: Compositions for the Delivery of Negatively Charged Molecules
Filed: April 14, 2005
Lead Inventor: Leonid Beigelman, Sirna Therapeutics (Merck)
"This invention features permeability-enhancer molecules and methods to increase membrane permeability of various molecules, such as nucleic acids, polynucleotides, oligonucleotides, enzymatic nucleic acid molecules, antisense nucleic acid molecules, 2-5A antisense chimeras, triplex forming oligonucleotides, decoy RNAS, dsRNAs, siRNAs, aptamers, or antisense nucleic acids containing nucleic acid cleaving chemical groups, peptides, polypeptides, proteins, carbohydrates, steroids, metals, and small molecules, thereby facilitating cellular uptake of such molecules," the patent's abstract states.
Title: Methods and Compositions for Use in Preparing siRNAs
Filed: April 30, 2003
Lead Inventor: Jason Myers, Stanford University
The patent, its abstract states, claims "methods and compositions for producing siRNAs — e.g., in the form of a dsiRNA composition — from dsRNAs are provided. In the subject methods, a dsRNA is contacted with a composition that includes an activity that cleaves dsRNA into siRNAs, where the composition efficiently cleaves dsRNA into siRNAs."
The siRNAs produced by the invention's methods "find use in a variety of applications, particularly in applications where the specific reduction or silencing of a gene is desired," the abstract notes. "Also provided are kits for use in practicing the subject invention."
Title: Methods and Compositions for Treating Brain Diseases
Filed: July 11, 2008
Lead Inventor: Beverly Davidson, University of Iowa
The patent application, its abstract states, claims "targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain."
These vectors, the application notes, can be used to deliver RNAi molecules.
Title: Novel siRNAs and Methods of Use
Filed: Oct. 25, 2007
Lead Inventor: Elena Feinstein, Quark Pharmaceuticals
"The invention relates to compounds, in particular siRNAs, which inhibit the expression of specific human genes," the patent application's abstract states. "The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier.
"The … invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject," it adds. "The invention also provides antibodies which inhibit specified human polypeptides and pharmaceutical compositions comprising one or more such antibodies."
Title: Pharmaceutical Compositions and Methods for Delivering Nucleic Acids into Cells
Filed: Nov. 24, 2008
Lead Inventor: Sang-Kyou Lee, ForHuman Tech
The invention, the patent application's abstract states, "relates to methods of delivering nucleic acids into cells using a nucleic acid binding molecule containing a multimeric or spacer-incorporated protein-transduction domain. The invention also relates to novel compositions that contain a nucleic acid complexed or conjugated with a nucleic acid binding molecule. The nucleic acid binding molecule may contain a multimeric or spacer-incorporated PTD, and may further contain a nucleic acid binding region. The nucleic acid complexes or conjugations of the … invention may be employed to inhibit expression of a target gene, and/or determine the function of a target gene," the abstract adds.
Title: Compositions and Methods for Modulation of PDX-1
Filed: Feb. 14, 2008
Lead Inventor: Irene Kazhdan, Ontherex
The invention, the patent application's abstract states, comprises "methods and compositions for inhibiting PDX-1. … An anti-PDX-1 agent included in inventive methods and compositions includes an antibody, an aptamer, an antisense oligonucleotide, a ribozyme, and/or an inhibitory compound.
"Methods of inhibiting PDX-1 expression in a tumor cell … include contacting a tumor cell with an effective amount of an anti-PDX-1 agent highly or completely complementary to a specified region of an RNA molecule encoding PDX-1," the abstract adds. "Such an agent specifically hybridizes with the RNA molecule encoding PDX-1 and inhibits the expression of a PDX-1 gene in the tumor cell. Compositions including anti-PDX-1 siRNA and/or shRNA are described … [as are] recombinant expression constructs encoding anti-PDX-1 siRNA or shRNA."
Title: Therapeutic Agent for Corneal Diseases
Filed: Nov. 8, 2006 PCT Filed: Nov. 8, 2006
Lead Inventor: Tetsuro Takamatsu, Kansai Technology Licensing Organization
The invention, the patent application's abstract states, "relates to a treatment agent for a disease or a disorder caused by a reduction in corneal endothelial cells, comprising as an active component at least one nucleic acid molecule, [namely an siRNA,] inhibiting the expression of a connexin 43 gene."
Title: miR-200-Regulated Genes and Pathways as Targets for Therapeutic Intervention
Filed: May 21, 2008
Lead Inventor: Andreas Bader, Mirna Therapeutics
According to the patent application's abstract, the invention "concerns methods and compositions for identifying genes or genetic pathways modulated by miR-200, using miR-200 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient, and/or treating the patient with an appropriate miRNA."
Title: Methods for Delivery of Nucleic Acids
Filed: Aug. 25, 2008
Lead Inventor: C. Satishchandran, Nucleonics (Alnylam Pharmaceuticals)
"This invention features methods and compositions for delivery of nucleic acids — e.g., DNA, RNA, PNA, and hybrids thereof — to cells," the patent application's abstract states. "The nucleic acid delivery complexes of the invention permit biologically active nucleic acids to be delivered to cells and organisms in vitro and in vivo in a manner and form that allows the nucleic acids to carry out their desired biological function."
Title: siRNA Targeting Tumor Necrosis Factor Receptor Superfamily Member 1A
Filed: Feb. 10, 2009
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
"Efficient sequence-specific gene silencing of siRNA targeting tumor necrosis factor receptor superfamily member 1A is possible through the use of siRNA technology," the patent application'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: siRNA Targeting Myeloid Cell Leukemia Sequence 1
Filed: Feb. 11, 2009
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
"Efficient sequence-specific gene silencing of myeloid cell leukemia sequence 1 is possible through the use of siRNA technology," the patent application's abstract states. "By selecting particular siRNAs directed to myeloid cell leukemia sequence 1 by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes."
Title: Cationic Lipids
Filed: May 20, 2008
Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals
The patent application, its abstract states, claims "cyclic lipid moieties."
Title: Modified Gene-Silencing RNA and Uses Thereof
Filed: July 18, 2008
Lead Inventor: Ming-Bo Wang, Commonwealth Scientific and Industrial Research Organization
The patent application, its abstract states, claims "methods and means for efficiently down-regulating the expression of any gene of interest in eukaryotic cells and organisms. … To this end, the invention provides modified antisense and sense RNA molecules, chimeric genes encoding such modified antisense or sense RNA molecules, and eukaryotic organisms such as plants, animals or fungi, yeast or molds, comprising the modified antisense and/or sense RNA molecules or the encoding chimeric genes."
Title: Compositions and Methods for Immunostimulatory RNA Oligonucleotides
Filed: Sept. 14, 2006 PCT Filed: Sept. 14, 2006
Lead Inventor: Gunther Hartmann, University of Bonn
The invention "provides 4-nucleotide RNA motifs that confer immunostimulatory activity, in particular, IFN-alpha-inducing activity, to a RNA oligonucleotide," the patent application's abstract states. "The … invention also provides RNA oligonucleotides, including siRNA, with high or low immunostimulatory activity … [as well as] the use of the RNA oligonucleotides of the invention for therapeutic purposes."
Title: Selected MA Motifs to Include Cell Death and/or Apoptosis
Filed: Nov. 14, 2008
Lead Inventor: Dan Smith, Multicell Immunotherapeutics
The patent application, its abstract states, is "directed to the use of dsRNA and/or ssRNA for the purpose of inducing apoptosis or cell death in proliferating cells. Specifically, low molecular-weight and high molecular-weight dsRNA and ssRNA are shown to induce apoptosis and/or cell death in proliferating cells, to arrest proliferation of transformed cells or tumor cells, and to cause rapid induction of the cytokine TNF-alpha and/or also induce production of IL-12, which directs a Th-1 response."
Title: Targeting of Tumor Stem Cells Through Selective Silencing of BORIS Expression
Filed: Nov. 10, 2008
Lead Inventor: Boris Reznik, OncoMune
The invention, the patent application's abstract states, "provides compositions useful for the treatment of cancer that inhibit tumor stem cells through suppression of an activity or the expression of BORIS. The compositions target tumor stem cells through molecules that are specific to tumor stem cells.
"Specifically, the invention provides immunoliposomes specific to tumor stem cells that include nucleic acid compositions capable of eliciting the process of RNA interference of BORIS expression," it notes. "Also provided are immunoliposomes specific to tumor stem cells that include anti-BORIS ribozymes, antisense oligonucleotides, decoy oligonucleotides, or small molecule inhibitors. Methods of manufacturing, delivering, and use of such compositions in the treatment of cancer are also provided."
Title: Inhibitors of Ribonucleotide Reductase Subunit 2 and Uses Thereof
Filed: Sept. 22, 2008
Lead Inventor: Mark Davis, Calando Pharmaceuticals (Arrowhead Research)
The patent application, its abstract states, "relates to inhibitors of ribonucleotide reductase subunit 2, and methods and compositions related to the R2 inhibitors. In certain embodiments, the R2 inhibitors include nucleic acids, such as for example siRNAs."
Title: Nucleic Acid-Mediated Treatment of Diseases or Conditions Related to Levels of Vascular Endothelial Growth Factor Receptor
Filed: July 9, 2008
Lead Inventor: Pamela Pavco, Sirna Therapeutics (Merck)
The invention "relates to nucleic acid molecules such as ribozymes, DNAzymes, short interfering RNA, short interfering nucleic acid, and antisense which modulate the synthesis, expression, and/or stability of an mRNA encoding one or more receptors of vascular endothelial growth factor, such as flt-1 and/or KDR," according to the patent application. "Nucleic acid molecules and methods for the inhibition of angiogenesis and treatment of cancer and other conditions associated with VEGF-R are provided, optionally in conjunction with other therapeutic agents such as interferons."
Title: Novel Cosmetic Designs and Products Using Intronic RNA
Filed: Dec. 28, 2007
Lead Inventor: Shi-Lung Lin, Mello Biotech Taiwan
The invention, the patent application's abstract states, "relates to a method and composition for generating a non-naturally occurring intron and its components capable of being processed into small hairpin RNA and/or microRNA (miRNA) molecules by skin cells and thus inducing specific gene-silencing effects on skin pigment-related genes and/or aging-causing genes in the cells. The gene silencing effects so obtained are not only useful for lightening and whitening skin colors, but also useful for suppressing unwanted aging gene activities in skins."
Title: Molecular Targets and Compounds, and Methods to Identify the Same, Useful in the Treatment of Joint Degenerative and Inflammatory Diseases
Filed: Jan. 27, 2009
Lead Inventor: Reginald Brys, Galapagos
The patent application, its abstract states, "discloses methods for identifying and using compounds that inhibit extra-cellular matrix degradation and inflammation using a [specific] polypeptide sequence … and fragments thereof, expression-inhibitory agents such as antisense polynucleotide, a ribozyme, and a small interfering RNA, comprising a nucleic acid sequence complementary to, or engineered from, a naturally occurring polynucleotide sequence encoding a [the aforementioned] polypeptide … [and] useful in pharmaceutical compositions comprising said agent, for the treatment, or prevention, of chronic joint degenerative and/or inflammatory diseases such as rheumatoid arthritis."
Title: Small Interfering RNAs that Efficiently Inhibit Viral Expression and Methods of Use Thereof
Filed: Sept. 12, 2005 PCT Filed: Sept. 12, 2005
Lead Inventor: Roger Kaspar, SomaGenics
"The invention provides methods, compositions, and kits comprising small interfering RNA … useful for inhibition of viral-mediated gene expression," the patent application's abstract states. "Small interfering RNAs as described herein may be used in methods of treatment of HCV infection … [and] shRNA and siRNA constructs that target the internal ribosome entry site sequence of HCV are described."
Title: Compositions and Methods for Efficient Gene Silencing in Plants
Filed: Oct. 19, 2007
Lead Inventor: Michael Axtell, Whitehead Institute for Biomedical Research
"This invention relates to methods for knockdown of a target gene in plants, particularly efficient and specific methods for knockdown of a target gene in plants," the patent application's abstract states. "This invention also relates to methods for silencing endogenous plant genes or plant pathogen genes … [and to] nucleic acid constructs [that] comprise a nucleic acid sequence that corresponds to a target gene or fragment thereof flanked by two complementary sites to an smRNA — e.g., a miRNA — resulting in, for example the configuration [of a] complementary site-nucleic acid sequence that corresponds to a target gene-complementary site."