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WO2016183366A3 - Compositions and methods for silencing expression of hepatitis d virus rna - Google Patents

Compositions and methods for silencing expression of hepatitis d virus rna Download PDF

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Publication number
WO2016183366A3
WO2016183366A3 PCT/US2016/032188 US2016032188W WO2016183366A3 WO 2016183366 A3 WO2016183366 A3 WO 2016183366A3 US 2016032188 W US2016032188 W US 2016032188W WO 2016183366 A3 WO2016183366 A3 WO 2016183366A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
hepatitis
compositions
virus rna
silencing expression
Prior art date
Application number
PCT/US2016/032188
Other languages
French (fr)
Other versions
WO2016183366A2 (en
Inventor
Amy C. H. Lee
Ian Maclachlan
Nicholas Michael SNEAD
Emily P. THI
Xin Ye
Original Assignee
Protiva Biotherapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Protiva Biotherapeutics, Inc. filed Critical Protiva Biotherapeutics, Inc.
Publication of WO2016183366A2 publication Critical patent/WO2016183366A2/en
Publication of WO2016183366A3 publication Critical patent/WO2016183366A3/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention provides compositions comprising therapeutic nucleic acids such as siRNA that target Hepatitis D virus (HDV) RNA expression, lipid particles comprising one or more (e.g., a combination) of the therapeutic nucleic acids, and methods of delivering and/or administering the lipid particles (e.g., for treating HDV infection in humans).
PCT/US2016/032188 2015-05-12 2016-05-12 Compositions and methods for silencing expression of hepatitis d virus rna WO2016183366A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562160411P 2015-05-12 2015-05-12
US62/160,411 2015-05-12
US201562269741P 2015-12-18 2015-12-18
US62/269,741 2015-12-18
US201662276072P 2016-01-07 2016-01-07
US62/276,072 2016-01-07

Publications (2)

Publication Number Publication Date
WO2016183366A2 WO2016183366A2 (en) 2016-11-17
WO2016183366A3 true WO2016183366A3 (en) 2016-12-29

Family

ID=57248513

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/032188 WO2016183366A2 (en) 2015-05-12 2016-05-12 Compositions and methods for silencing expression of hepatitis d virus rna

Country Status (1)

Country Link
WO (1) WO2016183366A2 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3586861T3 (en) 2011-06-08 2022-04-25 Translate Bio Inc LIPIDNANO PARTICLE COMPOSITIONS AND METHODS FOR MRNA SUBMISSION
BR112014030677A2 (en) 2012-06-08 2022-07-19 Shire Human Genetic Therapies pulmonary distribution of mrna to non-pulmonary target cells
PT2968586T (en) 2013-03-14 2018-11-13 Ethris Gmbh Cftr mrna compositions and related methods and uses
DK2970456T3 (en) 2013-03-14 2021-07-05 Translate Bio Inc METHODS AND COMPOSITIONS FOR DELIVERING MRNA-CODED ANTIBODIES
JP6586075B2 (en) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド Method for purifying messenger RNA
SI2972360T1 (en) 2013-03-15 2018-08-31 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
EA034103B1 (en) 2013-10-22 2019-12-27 Транслейт Био, Инк. METHOD OF TREATING PHENYLKETONURIA USING mRNA
EP3636742A1 (en) 2014-04-25 2020-04-15 Translate Bio, Inc. Methods for purification of messenger rna
ES2844180T3 (en) 2016-04-08 2021-07-21 Translate Bio Inc Multimeric encoding nucleic acid and uses thereof
AU2017283479B2 (en) 2016-06-13 2023-08-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
WO2018157154A2 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
MX2019013752A (en) 2017-05-16 2020-07-20 Translate Bio Inc Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr.
CA3084061A1 (en) 2017-12-20 2019-06-27 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
JP7432929B2 (en) * 2018-05-31 2024-02-19 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション RNA interference-inducing nucleic acids that suppress non-canonical targets of microRNAs and their uses
CN118421617A (en) 2018-08-24 2024-08-02 川斯勒佰尔公司 Method for purifying messenger RNA

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050058982A1 (en) * 2002-07-26 2005-03-17 Chiron Corporation Modified small interfering RNA molecules and methods of use
US20050170337A1 (en) * 1998-07-02 2005-08-04 President And Fellows Of Harvard College Oligomerization of hepatitis delta antigen
US20140248338A1 (en) * 2011-10-05 2014-09-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing aldehyde dehydrogenase
WO2015054451A1 (en) * 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050170337A1 (en) * 1998-07-02 2005-08-04 President And Fellows Of Harvard College Oligomerization of hepatitis delta antigen
US20050058982A1 (en) * 2002-07-26 2005-03-17 Chiron Corporation Modified small interfering RNA molecules and methods of use
US20140248338A1 (en) * 2011-10-05 2014-09-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing aldehyde dehydrogenase
WO2015054451A1 (en) * 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma

Also Published As

Publication number Publication date
WO2016183366A2 (en) 2016-11-17

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