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WO2016183366A3 - Compositions et méthodes permettant l'extinction de l'expression de l'arn du virus de l'hépatite d - Google Patents

Compositions et méthodes permettant l'extinction de l'expression de l'arn du virus de l'hépatite d 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
English (en)
Other versions
WO2016183366A2 (fr
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/fr
Publication of WO2016183366A3 publication Critical patent/WO2016183366A3/fr

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 nucleic acids [NA]
    • 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

La présente invention se rapporte à des compositions qui contiennent des acides nucléiques thérapeutiques, tels qu'un ARNsi, qui ciblent l'expression de l'ARN du virus de l'hépatite D (VHD), des particules lipidiques comprenant un ou plusieurs (par ex., une combinaison) desdits acides nucléiques thérapeutiques, et des méthodes de distribution et/ou d'administration des particules lipidiques (par ex., pour traiter l'infection par le VHD chez l'homme).
PCT/US2016/032188 2015-05-12 2016-05-12 Compositions et méthodes permettant l'extinction de l'expression de l'arn du virus de l'hépatite d WO2016183366A2 (fr)

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 (fr) 2016-11-17
WO2016183366A3 true WO2016183366A3 (fr) 2016-12-29

Family

ID=57248513

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/032188 WO2016183366A2 (fr) 2015-05-12 2016-05-12 Compositions et méthodes permettant l'extinction de l'expression de l'arn du virus de l'hépatite d

Country Status (1)

Country Link
WO (1) WO2016183366A2 (fr)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012170930A1 (fr) 2011-06-08 2012-12-13 Shire Human Genetic Therapies, Inc Compositions de nanoparticules lipides et procédés pour le transfert d'arnm
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
DK2970456T3 (da) 2013-03-14 2021-07-05 Translate Bio Inc Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
JP6586075B2 (ja) 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 メッセンジャーｒｎａの精製方法
EP3446712A1 (fr) 2013-03-14 2019-02-27 Translate Bio Ma, Inc. Compositions d'arnm cftr et procédés et utilisations associés
SI2972360T1 (en) 2013-03-15 2018-08-31 Translate Bio, Inc. Synergistic improvement in the delivery of nucleic acids via mixed formulations
EA201992208A1 (ru) 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
SG11201608725YA (en) 2014-04-25 2016-11-29 Shire Human Genetic Therapies Methods for purification of messenger rna
CN109072223B (zh) 2016-04-08 2022-10-21 川斯勒佰尔公司 多聚体编码核酸及其用途
WO2017218524A1 (fr) 2016-06-13 2017-12-21 Rana Therapeutics, Inc. Thérapie à base d'arn messager pour le traitement de la carence en ornithine transcarbamylase
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
CA3063531A1 (fr) 2017-05-16 2018-11-22 Translate Bio, Inc. Traitement de la fibrose kystique par administration d'arnm a codons optimises codant pour la cftr
CA3084061A1 (fr) 2017-12-20 2019-06-27 Translate Bio, Inc. Compositions et procedes ameliores pour le traitement du deficit en ornithine transcarbamylase
JP7432929B2 (ja) * 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 マイクロｒｎａの非正規標的を抑制するｒｎａ干渉誘導核酸およびその用途
EP3841208A1 (fr) 2018-08-24 2021-06-30 Translate Bio, Inc. Procédés de purification d'arn messager
EP3883592A1 (fr) 2018-11-21 2021-09-29 Translate Bio, Inc. Traitement de la fibrose kystique par administration d'arnm nébulisé codant pour la cftr

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 (fr) * 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Détection du virus de l'hépatite delta (vhd) pour le diagnostic et le traitement du syndrome de sjögren et du lymphome

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 (fr) * 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Détection du virus de l'hépatite delta (vhd) pour le diagnostic et le traitement du syndrome de sjögren et du lymphome

Also Published As

Publication number Publication date
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