Abstract: Methods for treating a neurodegenerative disorder associated with aggregation of tau protein in the brain of a subject, comprising administering to the subject a therapeutically effective amount of one or more inhibitory nucleic acids targeting microRNA-26a, microRNA-26b, or both microRNA-26a and 26b.
Abstract: RNA interference (RNAi) agents and RNAi agent conjugates for inhibiting the expression of the LPA (apo(a)) gene are described. Pharmaceutical compositions comprising one or more LPA RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described LPA RNAi agents to liver cells in vivo provides for inhibition of LPA gene expression and treatment of cardiovascular and cardiovascular-related diseases.
Type:
Grant
Filed:
September 30, 2016
Date of Patent:
April 3, 2018
Assignee:
Arrowhead Pharmaceuticals, Inc.
Inventors:
Stacey Melquist, Steven Kanner, David B. Rozema, David L. Lewis, Lauren J. Almeida, Darren H. Wakefield, Vladimir S. Trubetskoy, Tao Pei, Zhen Li, Aaron Almeida
Abstract: Compositions and methods for reducing susceptibility and enhancing tolerance to Nosema disease (Nosemosis) using RNA interference technology, and more particularly, prevention and treatment of Nosema infections in honeybees by feeding of Nosema-specific dsRNA.
Abstract: A method for determining concentrations of target proteins in a protein sample can involve: (i) contacting the protein sample with an aptamer library to form a mixture; (ii) allowing the aptamers in the aptamer library to bind to the target proteins in the protein sample; (iii) removing the aptamers that have not been bound to a target protein in the mixture; and (iv) measuring the concentration each aptamer bound to proteins in the mixture. The concentration of a particular protein in the protein sample can be derived from the measurements of the concentrations of the aptamer or aptamers bound to that particular protein in the mixture.
Abstract: Notch filter coatings for use in sunscreen applications are provided herein. An exemplary composition includes multiple zinc oxide particles suspended within a medium forming sunscreen composition; and a combination of multiple notch filter coating materials individually applied as a distinct layer to each of the multiple zinc oxide particles to create a multi-layered structure surrounding each of the multiple zinc oxide particles within the sunscreen composition, wherein the multi-layered structure: reflects light at a user-determined wavelength range based on the wavelength range at which each of the multiple notch filter coating materials reflects light; and allows wavelengths of light (i) within at least a portion of the ultraviolet spectrum and (ii) outside of the user-determined wavelength range to be absorbed by the multiple zinc oxide particles.
Type:
Grant
Filed:
March 3, 2017
Date of Patent:
February 13, 2018
Assignee:
International Business Machines Corporation
Inventors:
Talia S. Gershon, Ning Li, Devendra Sadana, Teodor K. Todorov
Abstract: The present invention provides a pharmaceutical agent which causes skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene with a high efficiency. The present invention provides an oligomer which efficiently enables to cause skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene.
Type:
Grant
Filed:
October 31, 2016
Date of Patent:
February 13, 2018
Assignees:
NIPPON SHINYAKU CO., LTD., NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
Inventors:
Naoki Watanabe, Haruna Seo, Shin'ichi Takeda, Tetsuya Nagata
Abstract: The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.
Type:
Grant
Filed:
May 20, 2016
Date of Patent:
February 6, 2018
Assignee:
Drexel University
Inventors:
Gordon J. Lutz, Melanie K. Tallent, Nicole Michele Lykens
Abstract: Notch filter coatings for use in sunscreen applications are provided herein. An exemplary composition includes multiple zinc oxide particles suspended within a medium forming sunscreen composition; and a combination of multiple notch filter coating materials individually applied as a distinct layer to each of the multiple zinc oxide particles to create a multi-layered structure surrounding each of the multiple zinc oxide particles within the sunscreen composition, wherein the multi-layered structure: reflects light at a user-determined wavelength range based on the wavelength range at which each of the multiple notch filter coating materials reflects light; and allows wavelengths of light (i) within at least a portion of the ultraviolet spectrum and (ii) outside of the user-determined wavelength range to be absorbed by the multiple zinc oxide particles.
Type:
Grant
Filed:
March 28, 2016
Date of Patent:
February 6, 2018
Assignee:
International Business Machines Corporation
Inventors:
Talia S. Gershon, Ning Li, Devendra Sadana, Teodor K. Todorov
Abstract: Disclosed herein is an isolated nucleic acid molecule comprising a first nucleic acid sequence 5?-ACCCTGCCGCCTGGACTCCGCCTGT-3? (SEQ ID NO: 22), or a functional variant thereof, operably linked to a second, heterologous nucleic acid sequence. The isolated nucleic acid molecule can be DNA (in an expression vector) and RNA (mRNA, shRNA, orncRNA). Also disclosed is a microvesicle comprising the nucleic acid molecule and a microvesicle preparation comprising the microvesicle. Also disclosed is an in vitro method of producing a microvesicle preparation enriched for a specific RNA sequence by transfecting cells with the nucleic acid sequence, and isolating microvesicles generated therefrom. Methods of delivering therapeutic RNA to a subject are also disclosed.
Type:
Grant
Filed:
January 17, 2013
Date of Patent:
January 30, 2018
Assignee:
THE GENERAL HOSPTIAL CORPORATION
Inventors:
Okay Saydam, Mehmet Fatih Bolukbasi, Arda Mizrak, Xandra O. Breakefield
Abstract: Provided herein are microRNAs that target transforming growth factor beta (TGF-?) receptors and attenuate pathways of fibrosis. In particular, microRNA-1343 reduces expression of TGFBR1 and TGFBR2, decreases TGF-? signaling, represses pathways of fibrosis, and treats fibrotic diseases.
Type:
Grant
Filed:
August 12, 2016
Date of Patent:
January 2, 2018
Assignee:
Ann & Robert H. Lurie Children's Hospital
Abstract: An object of the present invention is to develop and provide a method for efficiently and conveniently producing a nucleic acid aptamer, particularly, a DNA aptamer, having high specificity for and high binding activity against a target substance.
Abstract: The present invention relates to a therapeutic compound comprising: an agent that inhibits the activity of at least one gene selected from the group consisting of HIC1, FOXS1, CREB5, IRF7, POU2F2, STAT4, TCF4, and/or an agent that enhances the activity of at least one gene selected from the group consisting of MAF, MEOX2, SIX2.
Type:
Grant
Filed:
July 30, 2014
Date of Patent:
December 12, 2017
Assignees:
Centre National de la Recherche Scientifique-CNRS CNRS, INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), URGO RECHERCHE INNOVATION ET DEVELOPPEMENT, UNIVERSITÉ PARIS DIDEROT—PARIS 7, ECOLE NORMALE SUPERIEURE
Abstract: Disclosed herein is a modular composition comprising 1) an oligonucleotide; 2) one or more tetraGalNAc ligands of Formula (I), which may be the same or different; optionally, 3) one or more linkers, which may be the same or different; 4) one or more peptides independently selected from Table 3, which may be the same or different; and optionally, 5) one or more targeting ligands, solubilizing agents, pharmacokinetics enhancing agents, lipids, and/or masking agents.
Type:
Grant
Filed:
April 7, 2017
Date of Patent:
December 12, 2017
Assignee:
Sirna Therapeutics, Inc.
Inventors:
David Tellers, Steven L. Colletti, Vadim Dudkin, Jeffrey Aaronson, Aaron Momose, Thomas Joseph Tucker, Yu Yuan, Kathleen B. Calati, Lu Tian, Rubina G. Parmar, Anthony W. Shaw, Weimin Wang, Rachel Anne Storr, Marina Busuek, Robert A. Kowtoniuk
Abstract: The present invention relates to probes for use in the detection, imaging, diagnosis, targeting, treatment, etc. of cancers expressing the gastrin releasing peptide receptor (GRPR). For example, such probes may be molecules conjugated to detectable labels which are preferably moieties suitable for detection by gamma imaging and SPECT or by positron emission tomography (PET) or magnetic resonance imaging (MRI) or fluorescence spectroscopy or optical imaging methods.
Type:
Grant
Filed:
September 25, 2013
Date of Patent:
December 12, 2017
Assignee:
Advanced Accelerator Applications USA, Inc.
Inventors:
Theodosia Maina-Nock, Berthold Artur Nock, Marion de Jong Hendriks
Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be 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 the nucleotide sequences for AAT.
Type:
Grant
Filed:
November 9, 2015
Date of Patent:
December 12, 2017
Assignee:
Thermo Fisher Scientific Inc.
Inventors:
Anastasia Khvorova, Angela Reynolds, Devin Leake, William Marshall, Steven Read, Stephen Scaringe
Abstract: The present disclosure relates to RNA aptamers and uses thereof, in particular, aptamers which specifically bind to CD133 and which demonstrate superior tumor penetration.
Abstract: Compositions for inhibiting oligonucleotide activity in vitro or in vivo to a cell that are formulated with at least one oligonucleotide encapsulated in a lipid nanoparticle, methods of making, and methods of using the same are disclosed.
Abstract: Disclosed is a pharmaceutical combination formulation comprising a first discrete part containing amlodipine and rosuvastatin and a second discrete part containing losartan, which exhibits improved dissolution rate and stability. The inventive combination formulation comprising amlodipine, losartan and rosuvastatin having different action mechanisms from one another can be effectively used to prevent or treat a cardiovascular disorder. Designed to minimize an interaction among active ingredients, the pharmaceutical combination formulation exhibits excellent storage stability and dissolution rates of amlodipine, losartan and rosuvastatin, and thus can be useful in pharmaceutical industries.
Type:
Grant
Filed:
November 20, 2014
Date of Patent:
December 5, 2017
Assignee:
HANMI PHARM. CO., LTD
Inventors:
Ho Taek Im, Myoung Ki Jeong, Yong Il Kim, Jae Hyun Park, Jong Soo Woo, Hyuk Jun Cho
Abstract: Provided is a felbinac-containing external patch which can exhibit high drug release properties, low skin irritation, and high drug stability, wherein felbinac having an average particle diameter of 5 ?m or more and less than 100 ?m is dispersed and mixed in an adhesive base including a styrene-isoprene-styrene block copolymer, an alicyclic saturated hydrocarbon resin, a softener, and diethyl sebacate, and does not contain L-menthol. Specifically, in the felbinac-containing external patch, 0.1 to 10% by weight of felbinac having an average particle diameter of 5 ?m or more and less than 100 ?m is dispersed and mixed in an adhesive base including 10 to 30% by weight of a styrene-isoprene-styrene block copolymer, 10 to 50% by weight of an alicyclic saturated hydrocarbon resin, 10 to 75% by weight of a softener, and 0.1 to 10% by weight of diethyl sebacate.