Nucleic Acid Expression Inhibitors Patents (Class 536/24.5)
  • Patent number: 10392620
    Abstract: This disclosure concerns nucleic acid molecules and methods of use thereof for control of pathogens through RNA interference-mediated inhibition of target coding and transcribed non-coding sequences in pathogens. The disclosure also concerns methods for applying dsRNA through formulations and/or transgenic plants that express nucleic acid molecules useful for the control of pathogens, and the plant cells and plants obtained thereby.
    Type: Grant
    Filed: November 1, 2017
    Date of Patent: August 27, 2019
    Assignee: Dow AgroSciences LLC
    Inventors: Javier A. Delgado, Justin M. Lira, Chaoxian Geng, Meghan L. Frey
  • Patent number: 10370670
    Abstract: The invention provides a double-stranded ribonucleic acid (dsRNA) having a chain length suitable for simultaneously showing low toxicity and high function in the use of an adjuvant and the like, and resisting variation of chain length even when subjected to a heating and cooling treatment, or a salt thereof; an immune potentiator, adjuvant, pharmaceutical product and the like containing the dsRNA and the like; and a production method of such dsRNA and the like. The invention is characterized in that the weight average chain length of two or more single-stranded ribonucleic acids (ssRNAs) constituting the first chain constituting dsRNA is not more than ½ of the weight average chain length of one ssRNA constituting the second chain.
    Type: Grant
    Filed: October 24, 2017
    Date of Patent: August 6, 2019
    Assignee: KYOWA HAKKO BIO CO., LTD.
    Inventors: Tetsuo Nakano, Eitora Yamamura
  • Patent number: 10370659
    Abstract: In certain embodiments, the present disclosure provides compounds and methods for increasing the antisense activity of an antisense compound in a cell. In certain embodiments, the present disclosure provides methods for identifying antisense compounds having high activity. In certain embodiments, the present disclosure provides methods for identifying antisense compounds that bind to enhancer or repressor proteins.
    Type: Grant
    Filed: February 23, 2016
    Date of Patent: August 6, 2019
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Xue-hai Liang, Wen Shen, Stanley T. Crooke
  • Patent number: 10370661
    Abstract: Materials and methods for regulating gene expression using nanoparticles functionalized with antisense oligonucleotides are provided.
    Type: Grant
    Filed: July 19, 2017
    Date of Patent: August 6, 2019
    Assignee: NORTHWESTERN UNIVERSITY
    Inventors: Chad A. Mirkin, Nathaniel L. Rosi, C. Shad Thaxton, David A. Giljohann
  • Patent number: 10363266
    Abstract: The present invention relates to antisense-oligonucleotides having a length of at least 10 nucleotides, wherein at least two of the nucleotides are LNAs, their use as inhibitors of TGF-R signaling, pharmaceutical compositions containing such antisense-oligonucleotides and the use for prophylaxis and treatment of neurological, neurodegenerative, fibrotic and hyperproliferative diseases.
    Type: Grant
    Filed: November 16, 2015
    Date of Patent: July 30, 2019
    Assignee: NeuroVision Pharma GmbH
    Inventors: Markus Hossbach, Monika Krampert, Hans-Lothar Arth
  • Patent number: 10357509
    Abstract: The present disclosure relates to compositions and methods for treating cancers using antisense (AS) nucleic acids directed against Insulin-like Growth Factor 1 Receptor (IGF-1R). The AS may be administered to the patients systemically, or may be used to produce an autologous cancer cell vaccine. In embodiments, the AS are provided in an implantable irradiated biodiffusion chamber comprising tumor cells and an effective amount of the AS. The chambers are irradiated and implanted in the abdomen of subjects and stimulate an immune response that attacks tumors distally. The compositions and methods disclosed herein may be used to treat many different kinds of cancer, for example glioblastoma.
    Type: Grant
    Filed: March 9, 2018
    Date of Patent: July 23, 2019
    Assignee: Thomas Jefferson University
    Inventors: David W. Andrews, Douglas C. Hooper
  • Patent number: 10358648
    Abstract: In certain aspects, provided herein are RNA complexes (e.g., asymmetric RNA complexes, such as asiRNAs or cell penetrating asiRNAs) that inhibit IL4R?, TRPA1, and/or F2RL1 expression and are therefore useful for treating atopic dermatitis or asthma.
    Type: Grant
    Filed: February 1, 2017
    Date of Patent: July 23, 2019
    Assignee: OliX Pharmaceuticals, Inc.
    Inventors: Dong Ki Lee, Sun Woo Hong, Hanna Lee, Dayeon Yu, Ji Eom
  • Patent number: 10351855
    Abstract: The present invention relates to a pharmaceutical composition for inhibiting cancer cell metastasis, the pharmaceutical composition including at least one of: (1) si-rpS3/203 binding to a 203rd base sequence of rpS3 mRNA; (2) si-rpS3/635 binding to a base sequence corresponding to a position 635 of the rpS3 mRNA; (3) si-rpS3/747 binding to a base sequence corresponding to a position 747 of the rpS3 mRNA; and (4) si-rpS3/766 binding to a base sequence corresponding to a position 766 of the rpS3 mRNA.
    Type: Grant
    Filed: June 21, 2017
    Date of Patent: July 16, 2019
    Inventor: Joon Kim
  • Patent number: 10342758
    Abstract: Provided are a cationic lipid which facilitates the introduction of a nucleic acid into, for example, a cell or the like; a composition containing the cationic lipid and a nucleic acid; a method for introducing a nucleic acid into a cell by using a composition containing the cationic lipid and the nucleic acid; and the like. The cationic lipid is, for example, a cationic lipid represented by formula (A): formula (A): (wherein R1 is alkenyl or the like, R2 is alkenyl or the like, R3 and R4 are each alkyl, or are combined together to form alkylene, or R3 and R5 are combined together to form alkylene, R5 is a hydrogen atom or the like, or is combined together with R3 to form alkylene, X1 is alkylene, and X2 is a single bond or alkylene).
    Type: Grant
    Filed: July 8, 2013
    Date of Patent: July 9, 2019
    Assignee: KYOWA HAKKO KIRIN CO., LTD.
    Inventors: Takeshi Kuboyama, Tomohiro Era, Tomoyuki Naoi, Kaori Yagi, Shintaro Hosoe
  • Patent number: 10337016
    Abstract: The present disclosure provides a pharmaceutical composition for treating cancer comprising an RNA oligonucleotide having a particular sequence and structure. Specifically, when a cell line is treated with an RNA oligonucleotide having specific sequence and helical bend structure according to the present disclosure, the expression of ISG56 is increased and apoptosis of cancer cells is induced. Thus, a composition comprising the RNA oligonucleotide can be used as an anticancer agent.
    Type: Grant
    Filed: October 14, 2016
    Date of Patent: July 2, 2019
    Assignee: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Byong-Seok Choi, Suk-Jo Kang, Janghyun Lee, Ji Youn Min, Dongmin Chun, Si-Eun Sung
  • Patent number: 10337010
    Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
    Type: Grant
    Filed: August 23, 2017
    Date of Patent: July 2, 2019
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
  • Patent number: 10329568
    Abstract: The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.
    Type: Grant
    Filed: September 5, 2018
    Date of Patent: June 25, 2019
    Assignee: Silence Therapeutics GMBH
    Inventors: Klaus Giese, Jörg Kaufmann, Anke Klippel-Giese
  • Patent number: 10323246
    Abstract: The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.
    Type: Grant
    Filed: August 15, 2017
    Date of Patent: June 18, 2019
    Assignee: Silence Therapeutics GMBH
    Inventors: Klaus Giese, Jörg Kaufmann, Anke Klippel-Giese
  • Patent number: 10302644
    Abstract: The invention provides, inter alia, methods of prognosing the survival of a multiple myeloma patient based levels of TP53RK in multiple myeloma cells of the patient. Also provided are methods of screening for therapeutic agents for treating multiple myeloma based on their ability to decrease TP53RK levels or activity in a patient with multiple myeloma.
    Type: Grant
    Filed: October 20, 2015
    Date of Patent: May 28, 2019
    Assignee: Dana-Farber Cancer Institute, Inc.
    Inventors: Teru Hideshima, Kenneth C. Anderson, Francesca Cottini
  • Patent number: 10232055
    Abstract: This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
    Type: Grant
    Filed: October 31, 2016
    Date of Patent: March 19, 2019
    Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
    Inventors: Katalin Kariko, Drew Weissman
  • Patent number: 10219492
    Abstract: Provided herein are methods for altering respiratory syncytial virus (RSV) replication in a cell using oligonucleotides derived from tRNAs, also referred to as tRFs (tRNA-derived RNA Fragments). The oligonucleotides may be used to decrease or increase replication of RSV. Also provided herein are methods for treating a subject having or at risk of having an RSV infection, and animal models for evaluating viral and host factors in RSV pathogenesis.
    Type: Grant
    Filed: November 21, 2014
    Date of Patent: March 5, 2019
    Assignee: The Board of Regents of the University of Texas System
    Inventors: Xiaoyong Bao, Yong Sun Lee
  • Patent number: 10208309
    Abstract: The present invention relates to a novel siRNA, and a high-efficiency double-stranded oligo RNA structure containing the same, and a nanoparticle containing the high-efficiency double-stranded oligo RNA structure. The double-stranded oligo RNA structure has a structure in which a hydrophilic material and a hydrophobic material are conjugated to both ends of a double-stranded oligo RNA (siRNA) via a simple covalent bond or linker-mediated covalent bond in order to be efficiently delivered into cells, and may be converted into a nanoparticle form in an aqueous solution by hydrophobic interactions of double-stranded oligo RNA structures. It is preferable that the siRNA contained in the double-stranded oligo RNA structure is an siRNA specific for fibrosis or respiratory disease-related gene, particularly, amphiregulin or stratifin.
    Type: Grant
    Filed: April 6, 2015
    Date of Patent: February 19, 2019
    Assignee: BIONEER CORPORATION
    Inventors: Jeiwook Chae, Pyoung Oh Yoon, Boram Han, Mi Na Kim, Youngho Ko, Han Oh Park
  • Patent number: 10202599
    Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
    Type: Grant
    Filed: August 8, 2012
    Date of Patent: February 12, 2019
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Eric E. Swayze
  • Patent number: 10174327
    Abstract: The present disclosure provides an RNA oligonucleotide having a helical bend structure and a use thereof. Specifically, double strands formed by a complementary binding of two sequences have a helical bend structure in the RNA oligonucleotide. The RNA oligonucleotide can increase the expression of interferon-? or ISG56 and thus can be used as an immune system enhancer.
    Type: Grant
    Filed: May 26, 2016
    Date of Patent: January 8, 2019
    Assignee: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Byong-Seok Choi, Mi Kyung Lee, Janghyun Lee, Suk-Jo Kang, Eun Byeol Park
  • Patent number: 10138485
    Abstract: Neutral lipid formulations for nucleic acid delivery are provided according to the invention. The neutral lipid formulations include hydrophobically modified polynucleotides and fat mixtures. Methods of using the neutral lipid formulations are also provided.
    Type: Grant
    Filed: September 22, 2009
    Date of Patent: November 27, 2018
    Assignee: RXi Pharmaceuticals Corporation
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Michelle Miller, Karen G. Bulock
  • Patent number: 10131910
    Abstract: The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Usher syndrome type 2A and/or USH2A-associated non syndromic retina degeneration.
    Type: Grant
    Filed: July 9, 2015
    Date of Patent: November 20, 2018
    Assignee: STICHTING KATHOLIEKE UNIVERSITEIT
    Inventor: Hendrikus Antonius Rudolfus Van Wyk
  • Patent number: 10119136
    Abstract: One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene in vivo. The dsRNA duplex comprises one or more 4?-modifications in one or both strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for in vivo therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: January 9, 2015
    Date of Patent: November 6, 2018
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Muthiah Manoharan, Shigeo Matsuda, Kallanthottathil G. Rajeev, Ivan Zlatev
  • Patent number: 10106852
    Abstract: This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to the use of mutated ACVR1 in the diagnosis and treatment of Fibrodysplasia Ossificans Progressiva (FOP).
    Type: Grant
    Filed: October 22, 2014
    Date of Patent: October 23, 2018
    Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
    Inventors: Frederick S. Kaplan, Eileen M. Shore
  • Patent number: 10106792
    Abstract: The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.
    Type: Grant
    Filed: December 12, 2016
    Date of Patent: October 23, 2018
    Assignees: CITY OF HOPE, INTEGRATED DNA TECHNOLOGIES, INC.
    Inventors: John J. Rossi, Mark A. Behlke, Dongho Kim
  • Patent number: 10100004
    Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
    Type: Grant
    Filed: June 8, 2016
    Date of Patent: October 16, 2018
    Assignee: Nitto Denko Corporation
    Inventors: Yoshiro Niitsu, Joseph E. Payne, John A. Gaudette, Zheng Hou, Victor Knopov, Richard P. Witte, Mohammad Ahmadian, Loren A. Perelman, Yasunobu Tanaka, Violetta Akopian
  • Patent number: 10100310
    Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
    Type: Grant
    Filed: April 11, 2017
    Date of Patent: October 16, 2018
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Patent number: 10036022
    Abstract: The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.
    Type: Grant
    Filed: November 18, 2016
    Date of Patent: July 31, 2018
    Assignee: Nogra Pharma Limited
    Inventor: Giovanni Monteleone
  • Patent number: 10004814
    Abstract: The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
    Type: Grant
    Filed: November 10, 2014
    Date of Patent: June 26, 2018
    Assignee: SIRNA THERAPEUTICS, INC.
    Inventors: Marija Tadin-Strapps, Tayeba Khan, Walter Richard Strapps, Laura Sepp-Lorenzino, Vasant Jadhav, Duncan Brown
  • Patent number: 10000766
    Abstract: Provided is a strategy for generating transgenic plants with concurrent resistance to DNA and RNA viruses at one construction, so as to develop an RNA-directed DNA methylation (RdDM) transgenic system using a hairpin construct of Ageratum yellow vein virus (AYVV) promoter region residing in an intron to resist DNA virus infection by RdDM. Furthermore, the hairpin construct of the AYVV promoter region coupled with an untranslatable nucleocapsid protein (NP) fragment of Melon yellow sport virus (MYSV) is created to induce post-transcriptional gene silencing (PTGS) against MYSV. A method for providing transgenic plants conferring concurrent resistance to both AYVV and MYSV for control of DNA and RNA virus at the same time, and underlying RdDM and PTGS mechanisms, respectively, is also provided.
    Type: Grant
    Filed: July 17, 2015
    Date of Patent: June 19, 2018
    Assignee: NATIONAL CHUNG HSING UNIVERSITY
    Inventors: Shyi-Dong Yeh, Ching-Fu Yang, Kuan-Chun Chen, Ya-Ling Huang
  • Patent number: 9999772
    Abstract: A system and method for performing deep brain stimulation (DBS) therapy are provided. The method and system include pre-operatively acquiring at least one pre-operative image of the brain with at least one imaging sub-system and determining a location of a Nucleus Basalis of Meynert (NBM) for therapy in the at least one pre-operative image, and intra-operatively acquiring at least one intra-operative image of the brain after obtaining an access opening through the skull. The method and system further provide performing surgical planning based on the pre-operative image in the intra-operative image, advancing a lead having DBS electrodes on the lead to a target position proximate to or within the NBM area, and coupling the lead to an implantable pulse generator (IPG) configured to deliver DBS pulses through the DBS electrodes to the NBM. Further, the IPG is configured to deliver DBS pulses for treating symptoms associated with Alzheimer's disease.
    Type: Grant
    Filed: April 3, 2014
    Date of Patent: June 19, 2018
    Assignee: PACESETTER, INC.
    Inventor: Lalit Venkatesan
  • Patent number: 9982266
    Abstract: The present invention relates to a method and compositions for the treatment of cystic fibrosis.
    Type: Grant
    Filed: May 17, 2017
    Date of Patent: May 29, 2018
    Assignees: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE DE MONTPELLIER
    Inventor: Magali Taulan-Cadars
  • Patent number: 9944931
    Abstract: The present disclosure provides, in part, methods of discovering immunotherapy targets in vivo, therapeutic compositions (e.g., shRNA, immunoresponsive cells expressing shRNA and/or a chimeric antigen receptors (CAR)), and methods of use thereof.
    Type: Grant
    Filed: June 10, 2014
    Date of Patent: April 17, 2018
    Assignees: Dana-Farber Cancer Institute, Inc., The General Hospital Corporation
    Inventors: Kai W. Wucherpfennig, Glenn Dranoff, Penghui Zhou, Donald Shaffer, Nir Hacohen, Harvey I. Cantor, Diana Alvarez Arias
  • Patent number: 9944942
    Abstract: The present invention provides transgenic plants having increased tolerance to abiotic stress comprising a recombinant nucleic acid molecule, said recombinant nucleic acid molecule comprising a nucleotide sequence encoding miR319 operatively associated with a promoter, a nucleotide sequence that is antisense to a portion of consecutive nucleotides of a nucleotide sequence encoding PCF5, and/or a nucleotide sequence that encodes a portion of consecutive nucleotides of a nucleotide sequence encoding PCF5, which when expressed produces an antisense nucleotide sequence, wherein expression of the nucleotide sequence confers increased tolerance to abiotic stress. Also provided are methods and compositions for making said transgenic plants.
    Type: Grant
    Filed: November 8, 2012
    Date of Patent: April 17, 2018
    Assignee: CLEMSON UNIVERSITY
    Inventors: Hong Luo, Dayong Li, Man Zhou, Qian Hu
  • Patent number: 9914922
    Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
    Type: Grant
    Filed: April 22, 2013
    Date of Patent: March 13, 2018
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Eric E. Swayze
  • Patent number: 9868949
    Abstract: The present disclosure relates to methods of treating EPAS1-related diseases such as cancer, metastases, astrocytoma, bladder cancer, breast cancer, chondrosarcoma, colorectal carcinoma, gastric carcinoma, glioblastoma, head and neck squamous cell carcinoma, hepatocellular carcinoma, lung adenocarcinoma, neuroblastoma, non-small cell lung cancer, melanoma, multiple myeloma, ovarian cancer, rectal cancer, renal cancer, clear cell renal cell carcinoma (and metastases of this and other cancers), gingivitis, psoriasis, Kaposi's sarcoma-associated herpesvirus, preemclampsia, inflammation, chronic inflammation, neovascular diseases, and rheumatoid arthritis, using a therapeutically effective amount of a RNAi agent to EPAS1.
    Type: Grant
    Filed: February 27, 2014
    Date of Patent: January 16, 2018
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: Brian Bettencourt, Shanthi Ganesh, Elizabeth George, Dieter Heusken, Stuart Milstein, Jonathan Solomon, Emily Thomas, Ivanka Toudjarska, Jennifer Tullai, Jan Weiler
  • Patent number: 9868782
    Abstract: This invention features methods and compositions useful for treating and diagnosing diseases of the nervous system, retina, skin, muscle, joint, and cartilage using a Dragon family protein. Protein and nucleic acid sequences of human, murine, zebrafish, and C. elegans Dragon family members are also disclosed.
    Type: Grant
    Filed: October 26, 2011
    Date of Patent: January 16, 2018
    Assignee: The General Hospital Corporation
    Inventors: Clifford J. Woolf, Tarek A. Samad
  • Patent number: 9856480
    Abstract: The invention provides DNAzymes which are capable to silence the expression of EGFR at allele-specific level. These allele-specific DNAzymes against EGFR T790M mutation will knockdown the expression of EGFR T790M mRNA while keeping EGFR wild-type mRNA intact. Hence, these allele-specific DNAzymes against EGFR T790M mutation may overcome T790M-derived TKI resistance accompanied with lower unwanted side effects on normal cells in lung cancer patients.
    Type: Grant
    Filed: January 14, 2014
    Date of Patent: January 2, 2018
    Assignees: National Taiwan University, ACADEMIA SINICA
    Inventors: Pan-Chyr Yang, Wei-Yun Lai, Konan Peck, Cheng-Ju Chang, Chi-Yuan Chen, Shuenn-Chen Yang
  • Patent number: 9816092
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.
    Type: Grant
    Filed: June 23, 2016
    Date of Patent: November 14, 2017
    Assignee: Ionis Phamaceuticals, Inc.
    Inventors: Brett P. Monia, Susan M. Freier, Andrew M. Siwkowski, Shuling Guo
  • Patent number: 9796974
    Abstract: One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: November 16, 2012
    Date of Patent: October 24, 2017
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Kallanthottathil G. Rajeev, Tracy Zimmermann, Muthiah Manoharan, Martin Maier, Satyanarayana Kuchimanchi, Klaus Charisse
  • Patent number: 9790494
    Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.
    Type: Grant
    Filed: April 28, 2015
    Date of Patent: October 17, 2017
    Assignee: Translate Bio MA, Inc.
    Inventors: Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
  • Patent number: 9777272
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
    Type: Grant
    Filed: September 16, 2016
    Date of Patent: October 3, 2017
    Assignees: INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP—Chimie ParisTech—Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
    Inventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
  • Patent number: 9771585
    Abstract: The present invention provides oligonucleotide inhibitors of miR-155 and compositions thereof. The invention further provides methods for treating cancer such as a T cell lymphoma in a subject by administering to the subject an oligonucleotide inhibitor of miR-155. The invention also provides methods for reducing or inhibiting the proliferation of malignant T cells by administering an oligonucleotide inhibitor of miR-155.
    Type: Grant
    Filed: June 3, 2016
    Date of Patent: September 26, 2017
    Assignee: MIRAGEN THERAPEUTICS, INC.
    Inventors: David Rodman, Anita Seto, Christina Dalby, Aimee Jackson, Xuan Beatty
  • Patent number: 9745369
    Abstract: The present invention relates to compounds for use in modulating the toll-like receptor 4 (TLR4) signaling pathway, as well as to a pharmaceutical composition comprising said compounds.
    Type: Grant
    Filed: May 22, 2013
    Date of Patent: August 29, 2017
    Assignee: Rheinisch-Westfalische Technische
    Inventors: Antje Ostareck-Lederer, Dirk Ostareck, Gernot Marx, Anke Liepelt, Jana Kloos
  • Patent number: 9732348
    Abstract: The invention relates to a DNA aptamer that can inhibit the enzymatic activity of tyrosinase for the conversion of tyrosine into L-DOPA and dopaquinone, and to the dermatological and cosmetic uses thereof.
    Type: Grant
    Filed: December 20, 2013
    Date of Patent: August 15, 2017
    Assignees: L V M H Recherché, Institut National de la Sante et de la Recherche Medicale (INSERM)
    Inventors: Jean Hubert Cauchard, Robin Kurfurst, Sylvianne Schnebert, Eric Dausse, Jean-Jacques Toulme
  • Patent number: 9732340
    Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.
    Type: Grant
    Filed: April 28, 2015
    Date of Patent: August 15, 2017
    Assignee: Translate Bio MA, Inc.
    Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
  • Patent number: 9670492
    Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
    Type: Grant
    Filed: August 28, 2014
    Date of Patent: June 6, 2017
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Patent number: 9650638
    Abstract: This disclosure relates to DNA aptamer specifically binding to periostin, which is cancer-related protein, a composition for inhibiting cancer and/or cancer metastasis and/or a composition for diagnosis of cancer and/or cancer metastasis, comprising the same as an active ingredient, wherein the aptamer has different binding mechanism from the existing antibodies, and thus, may inhibit and diagnose cancer/cancer metastasis more effectively.
    Type: Grant
    Filed: November 12, 2012
    Date of Patent: May 16, 2017
    Assignees: UNIST (ULSAN NATIONAL INSTITUTE OF SCIENCE AND TECHNOLOGY), POSTECH ACADEMY-INDUSTRY FOUNDATION
    Inventors: Pann-Ghill Suh, Ii Shin Kim, Yu Jin Lee, Youn Dong Kim, Young Chan Chae, Jong Hun Im, Sung Ho Ryu
  • Patent number: 9642873
    Abstract: The present invention provides compositions and methods for using combinations of TGF?1 and Cox-2 inhibitors and TGF?1 and Hoxb13 inhibitors for the treatment of various medical conditions, including skin scaring due to trauma wounds and surgery, corneal and retina scaring due to injury and surgery, internal organ scaring due to injury and surgery, heart tissue scaring due to heart attack and surgery, and lung, liver, and kidney fibrosis due to inflammation and injury. One example is to use siRNA inhibitors to silence TGF?1 and Cox-2 at the same time, resulting in significant less scar formation.
    Type: Grant
    Filed: May 4, 2011
    Date of Patent: May 9, 2017
    Assignee: Sirnaomics, Inc.
    Inventors: Patrick Y. Lu, Vera Simonenko, David Evans, John J. Xu
  • Patent number: 9629867
    Abstract: Chemically modified small interfering RNAs (siRNAs) that include both phosphorodithioate modifications (PS2-RNA) and 2?-O-methyl modifications (MePS2) provide improved RNA silencing. Specific chemically modified siRNA that show enhanced silencing of RNAs involved in resistance to chemotherapeutic agents are provided.
    Type: Grant
    Filed: March 11, 2015
    Date of Patent: April 25, 2017
    Assignee: AM Biotechnologies, Inc.
    Inventor: Xianbin Yang
  • Patent number: RE46873
    Abstract: The present invention provides small interfering RNA (siRNA) molecules, compositions containing them, and methods of using them for improvement of skin scarless wound healing and other skin conditions, such as psoriasis and lupus-caused cutaneous lesions. The invention includes siRNA molecules and compositions containing them that inhibit the expression of one or more genes that promote pathological or undesired processes in wound healing and methods of using them.
    Type: Grant
    Filed: May 26, 2016
    Date of Patent: May 29, 2018
    Assignee: Sirnaomics, Inc.
    Inventors: Patrick Y. Lu, Ling Li, Vera Simonenko