Abstract: Methods and compositions for the amplification of nucleic acids and generation of concatemers are disclosed. Amplification methods provided herein may be performed under isothermal conditions. Methods and compositions may include reagents such nucleic acid polymerases and primers.
Abstract: This invention relates to compounds, compositions, and methods useful for reducing lactact dehydrogenase target RNA and protein levels via use of ds RNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Type:
Grant
Filed:
March 15, 2019
Date of Patent:
August 11, 2020
Assignee:
Dicerna Pharmaceuticals, Inc.
Inventors:
Bob D. Brown, Henryk T. Dudek, Cheng Lai
Abstract: Described herein are in vitro-transcribed (IVT) RNA molecules comprising, a 5? cap structure, a coding region encoding an antigen polypeptide, an immunostimulatory RNA sequence, and a poly(A) tail.
Type:
Grant
Filed:
October 4, 2018
Date of Patent:
August 11, 2020
Assignee:
President and Fellows of Harvard College
Inventors:
Ying Kai Chan, Jessica Jing-Shiuan Chiang
Abstract: The presently disclosed subject matter provides a novel approach for the treatment, prevention, and diagnosis of Cap-Snatching virus infections, particularly all classes of human influenza, including pandemic influenza. The methods involve the use of constructs for RNA-interference (RNAi).
Type:
Grant
Filed:
March 11, 2014
Date of Patent:
August 4, 2020
Assignee:
The Johns Hopkins University
Inventors:
Christopher E. Bradburne, Lucy M. Carruth
Abstract: Methods and materials for improved in vivo production of dsRNA are presented. Yields of dsRNA are significantly increased in the presence of capsid protein. The improved yield of dsRNA is not dependent on the presence of specific cognate binding sites for capsid protein associated with the dsRNA, but is dependent on capsid protein.
Type:
Grant
Filed:
March 9, 2017
Date of Patent:
July 7, 2020
Assignee:
APSE, INC.
Inventors:
John L. Killmer, Patrick D. McLaughlin, Juan Pedro Humberto Arhancet
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:
August 30, 2018
Date of Patent:
July 7, 2020
Assignee:
Translate Bio MA, Inc.
Inventors:
Eugen Uhlmann, Markus Weber, Romesh R. Subramanian, Thomas Dino Rockel, Arthur M. Krieg
Abstract: This invention is intended to enhance and improve the resistance of a single- or double-stranded nucleic acid fragment comprising a base sequence of a functional nucleic acid to degradation by nucleolytic enzymes in a simple and cost-effective manner. The single- or double-stranded nucleic acid fragment comprises, ligated to at least one end thereof, hairpin-shaped DNA comprising: (A) a nucleic acid region comprising 2 to 5 arbitrary nucleotides; (B) a nucleic acid region comprising a “gna” or “gnna” base sequence, wherein each “n” represents “g”, “t”, “a”, or “c”, a base analogue, or a modified base; and (C) a nucleic acid region comprising a base sequence complementary to the nucleic acid region (A), sequentially from the 5? end toward the 3? end.
Abstract: The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
Type:
Grant
Filed:
December 27, 2017
Date of Patent:
June 23, 2020
Assignee:
BioMarin Technologies B.V.
Inventors:
Josephus Johannes De Kimpe, Gerard Johannes Platenburg, Derick Gert Wansink
Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.
Type:
Grant
Filed:
August 22, 2018
Date of Patent:
June 16, 2020
Assignee:
Arcturus Therapeutics, Inc.
Inventors:
Kiyoshi Tachikawa, Joseph E. Payne, Padmanabh Chivukula
Abstract: The present disclosure relates to an inhibitor of Dynamin 2 for use in the treatment of centronuclear myopathies. The present disclosure relates to pharmaceutical compositions containing Dynamin 2 inhibitor and to their use for the treatment of centronuclear myopathies. It also deals with a method for identifying or screening molecules useful in the treatment of a centronuclear myopathy.
Type:
Grant
Filed:
October 20, 2014
Date of Patent:
May 12, 2020
Assignees:
UNIVERSITE DE STRASBOURG, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Abstract: Provided is a composition comprising an RNAi molecule which suppresses expression of CHST15 gene and successfully used for less invasive administration. A complex comprising an RNAi molecule which suppresses expression of CHST15 gene and an N-ace.
Abstract: The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.
Type:
Grant
Filed:
November 15, 2018
Date of Patent:
May 12, 2020
Assignee:
ModernaTX, Inc.
Inventors:
Joshua Frederick, Susannah Hewitt, Ailin Bai, Stephen Hoge, Vladimir Presnyak, Iain Mcfadyen, Kerry Benenato, Ellalahewage Sathyajith Kumarasinghe
Abstract: The present invention relates to a composition for reducing sweating in humans, characterized in that said composition comprises a compound capable of reduction of ITPR2 protein function and reduction of levels of ITPR2 mRNA and/or ITPR2 protein, and optionally pharmaceutically acceptable carriers and/or excipients, as well as to methods of treatment and specific siRNA molecules and their use in therapy.
Abstract: The present invention relates to stable low-salt aqueous formulations of particles comprising a polycation, preferably protamine, and RNA, which formulations are preferably isotonic, to pharmaceutical compositions or kits comprising such formulations and to their use in medicine. It further relates to methods for preparing the formulations of the invention and to methods for physically stabilizing particles comprising a polycation, preferably protamine, and RNA.
Abstract: Disclosed are methods and compositions for using microRNA (miRNA) for treating cancer. The methods and compositions include hsa-miR-204 and homologs of hsa-miR-204.
Type:
Grant
Filed:
August 14, 2017
Date of Patent:
April 14, 2020
Assignee:
THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
Inventors:
Manjeet K. Rao, Jaafer S. Imam, Behyar Zoghi, Yao-Fu Chang, Panneerdoss Subbarayalu
Abstract: The present invention relates to a pharmaceutical composition for treatment of cancer, which comprises, as an active ingredient, one or more miRNAs selected from the group consisting of miR-3670, miR-8078, and miR-4477a. The pharmaceutical composition for treatment of cancer according to the present invention exhibits excellent effects of inhibiting cancer cell proliferation and inducing cancer cell apoptosis. Thus, the pharmaceutical composition of the present invention can be effectively used as an anticancer therapeutic agent.
Type:
Grant
Filed:
March 31, 2019
Date of Patent:
April 7, 2020
Assignee:
BIONEER CORPORATION
Inventors:
Taewoo Lee, Sanghyung Shim, Ungsik Yu, Han Oh Park
Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.
Type:
Grant
Filed:
December 31, 2017
Date of Patent:
March 31, 2020
Assignee:
ARCTURUS THERAPEUTICS, INC.
Inventors:
Kiyoshi Tachikawa, Joseph E. Payne, Padmanabh Chivukula
Abstract: Disclosed herein are compounds, compositions and methods for modulating the amount or activity of a target nucleic acid. In certain embodiments, the amount or activity of a target nucleic acid is modulated through nonsense mediated decay.
Abstract: Methods and compositions are provided for modulating, e.g., reducing, viral coding sequence expression in mammals and mammalian cells. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.
Type:
Grant
Filed:
September 27, 2002
Date of Patent:
March 17, 2020
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Abstract: A double-stranded nucleic acid molecule for suppressing expression of non-coding RNA, the double-stranded nucleic acid molecule including: (a) a sense strand containing a nucleotide sequence corresponding to a target sequence set forth in any one of SEQ ID NOs: 5 to 11 and 26 to 31; and (b) an antisense strand containing a nucleotide sequence which is complementary to the sense strand in the (a) to form a double strand with the sense strand, wherein the non-coding RNA contains a base sequence set forth in any one of SEQ ID NOs: 1 to 4, a part of the base sequence, or both of the base sequence and the part.