Title: Polyconjugates for In Vivo Delivery of Polynucleotides
Patent Number: 8,137,695
Filed: Aug. 17, 2007
Lead Inventor: David Rozema, Roche (Arrowhead Research)
The invention, the patent's abstract states, “is directed to compounds, compositions, and methods useful for delivering polynucleotides or other cell-impermeable molecules to mammalian cells. Described are polyconjugate systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery vehicles. The use of multiple reversible linkages connecting component parts provides for physiologically responsive activity modulation.”
Title: Nanoparticles for Protein/Peptide Delivery and Delivery Means Thereof
Patent Number: 8,137,697
Filed: March 13, 2009
Lead Inventor: Hsing-Wen Sung, National Tsing Hua University
The invention, the patent's abstract states, comprises a “biodegradable nanoparticle for use in nanoparticle projectile bombardment as a carrier for administering nucleic acid, DNA, RNA, or siRNAs transdermally to an animal subject. The nanoparticles are composed of positively charged chitosan substrate and the nucleic acid encapsulated within the nanoparticles or electrostatically loaded on the exterior surface of the nanoparticles.”
Title: Orthotopic and Genetically Tractable Non-Human Animal Model for Liver Cancer and the Uses Thereof
Patent Number: 8,137,907
Filed: Feb. 21, 2008
Lead Inventor: Lars Zender, Cold Spring Harbor Laboratory
“This invention provides a genetically tractable in situ non-human animal model for hepatocellular carcinoma,” the patent's abstract states. “This invention also relates to the use of RNA interference technology in vivo to efficiently identify genes associated with liver cancer, in particular those encoding tumor suppressors, by knocking out candidate genes using RNAi and observing whether tumors would develop.”
Title: Method of Regulating Gene Expression
Patent Number: 8,137,910
Filed: May 5, 2003
Lead Inventor: Bryan Cullen, Duke University
The invention, the patent's abstract states, “relates, in general, to gene expression and, in particular, to a method of inhibiting the expression of a target gene and to constructs suitable for use in such a method.”
Title: Modified Small Interfering RNA Molecules and Methods of Use
Patent Number: 8,138,161
Filed: Sept. 30, 2005
Lead Inventor: Jang Han, Novartis
The invention comprises “double-stranded RNA molecules that mediate RNA interference in target cells, preferably hepatic cells,” according to the patent's abstract. “The invention also provides double-stranded RNA molecules that are modified to be resistant to nuclease degradation, which inactivates a virus, and more specifically, hepatitis C virus. The invention also provides a method of using these modified RNA molecules to inactivate virus in mammalian cells and a method of making modified small interfering RNAs using human Dicer.
“The invention provides modified RNA molecules that are modified to include a dsRNA or siRNA wherein one or more of the pyrimidines in the RNA molecule are modified to include 2'-fluorine,” the abstract adds. “The invention also provides dsRNA or siRNA in which all pyrimidines are modified to include a 2'-fluorine. The invention provides that the 2'-fluorine dsRNA or siRNA molecule is further modified to include a two base deoxynucleotide TT sequence at the 3' end of the molecule.”
Title: Inducible Systems and Methods for Controlling siRNA Expression
Patent Number: 8,138,327
Filed: Nov. 21 2005
Lead Inventor: Hoshang Unwalla, City of Hope
The patent, its abstract states, claims “an inducible system and methods for controlling expression of siRNA. ... An inducible system for producing siRNA only in the presence of HIV TAT, and methods for inhibiting HIV-1 gene expression in cells comprising such inducible system also are provided.”
Title: siRNA Targeting Connective Tissue Growth Factor
Patent Number: 8,138,329
Filed: Oct. 1, 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. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CTGF.”
Title: Method of Predicting Chemotherapy Effectiveness in Colon Adenocarcinoma Patients Using miR-21
Application Number: 20120065098
Filed: Nov. 15, 2011
Lead Inventor: Carlo Croce, Ohio State University
The invention, the patent application's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring [miRNA] levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”
Title: Methods and Compositions for the Treatment of Cancer or Other Diseases
Application Number: 20120065125
Filed: Sept. 9, 2011
Lead Inventor: Hua Yu, City of Hope
The invention, the patent application's abstract states, “relates to methods and compositions for the treatment of diseases, including cancer, infectious diseases and autoimmune diseases … [as well as] methods and compositions for improving immune function. More particularly, the ... invention relates to multifunctional molecules that are capable of being delivered to cells of interest for the treatment of diseases and for the improvement in immune function.”
Title: Lipids, Lipid Complexes, and Use Thereof
Application Number: 20120065138
Filed: Sept. 12, 2011
Lead Inventor: Oliver Keil, Silence Therapeutics
The invention relates to a compound of a pre-defined formula that is capable of delivering a pharmaceutical agent into cells, namely endothelial cells, epithelial cells, and tumor cells, according to the patent application.
Title: Polymeric Nanoparticles for siRNA Delivery Using Charge Interaction and Covalent Bonding
Application Number: 20120065242
Filed: Oct. 15, 2010
Lead Inventor: Kwang Meyung Kim, Korea Institute of Science and Technology
The patent application, its abstract states, claims a “stable in vivo polymer-siRNA delivery carrier in which a polymer and an siRNA are combined by using charge interaction and biodegradable covalent bonding at the same time and the use thereof. The polymer-siRNA delivery carrier … has a high siRNA delivery efficiency to a target portion in vivo. Hence, according to the polymer-siRNA binder, the siRNA for treatment can be effectively delivered to a target portion such as in vivo cancer tissue and the like even with administration of a relatively low concentration, and thus widely used for the treatment of various kinds of diseases.”
Title: RNA Duplexes with Single-Stranded Phosphorothioate Nucleotide Regions for Additional Functionality
Application Number: 20120065243
Filed: Feb. 4, 2010
Lead Inventor: Tod Woolf, RXi Pharmaceuticals
“The invention relates to RNAi constructs and their use in gene silencing,” the patent application's abstract states. “RNAi constructs associated with the invention contain a double-stranded region connected to a single-stranded region of phosphorothioate modified nucleotides.”
Title: Regulation of Hematopoietic Stem Cell Functions Through microRNAs
Application Number: 20120065245
Filed: Sept. 12, 2011
Lead Inventor: David Baltimore, California Institute of Technology
The invention, the patent application's abstract states, “relates to regulation of functions of hematopoietic stem cells by delivery of miRNAs, including miR-125b, miR-126, and miR-155, to HSCs. For example, in some embodiments, blood output in a mammal can be increased by administering miR-125b, miR-126, and/or miR-155 oligonucleotides. Also disclosed are methods for promoting hematopoietic stem cell engraftment and method for treating a myeloproliferative disorder.”
Title: RNA Molecules and Therapeutics Uses Thereof
Application Number: 20120065246
Filed: March 1, 2010
Inventor: Pal Saetrom, Mina Therapeutics
“The invention relates to double-stranded RNA molecules in which each strand of said molecule possesses sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof and sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex,” the patent application's abstract states. “At least one strand of said molecule possesses a seed region of complementarity to at least one seed site present in a 3' untranslated region of at least one target mRNA molecule.
“The invention also relates to an algorithm for the design of a double-stranded RNA molecule,” it adds.
Title: Methods and Composition Involving miRNA and miRNA Inhibitor Molecules
Application Number: 20120065248
Filed: Nov. 17, 2011
Lead Inventor: David Brown, Asuragen
The invention, the patent application's abstract states, “concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the ... invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.”
Title: siRNA Targeting Apolipoprotein B
Application Number: 20120065250
Filed: Oct. 27, 2011
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing 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. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for ApoB.”
Title: Biodegradable Poly-Beta-Amino Esters and Uses Thereof
Application Number: 20120065358
Filed: Nov. 21, 2011
Lead Inventor: Robert Langer, Massachusetts Institute of Technology
The invention, the patent application's abstract states, comprises “poly-beta-amino esters prepared from the conjugate addition of bis-secondary amines or primary amines to a bis-acrylate ester," and methods of preparing these polymers from commercially available starting materials.
“These tertiary amine-containing polymers are preferably biodegradable and biocompatible and may be used in a variety of drug delivery systems,” the abstract adds. “Given the polyamine nature of these polymers, they are particularly suited for the delivery of polynucleotides. Nanoparticles containing polymer/polynucleotide complexes have been prepared. The inventive polymers may also be used to encapsulate other agents to be delivered. They are particularly useful in delivering labile agents given their ability to buffer the pH of their surroundings.”