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WO2015042564A8 - Méthodes pour le traitement ou la prévention de maladies associées à la transthyrétine (ttr) - Google Patents

Méthodes pour le traitement ou la prévention de maladies associées à la transthyrétine (ttr) Download PDF

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Publication number
WO2015042564A8
WO2015042564A8 PCT/US2014/056923 US2014056923W WO2015042564A8 WO 2015042564 A8 WO2015042564 A8 WO 2015042564A8 US 2014056923 W US2014056923 W US 2014056923W WO 2015042564 A8 WO2015042564 A8 WO 2015042564A8
Authority
WO
WIPO (PCT)
Prior art keywords
ttr
treating
associated diseases
methods
transthyretin
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2014/056923
Other languages
English (en)
Other versions
WO2015042564A1 (fr
Inventor
Kallanthottathil G. Rajeev
Tracy Zimmermann
Muthiah Manoharan
Martin Maier
Satyanarayana Kuchimanchi
Klaus CHARISSE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of WO2015042564A1 publication Critical patent/WO2015042564A1/fr
Publication of WO2015042564A8 publication Critical patent/WO2015042564A8/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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Classifications

    • 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
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

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

Abstract

La présente invention concerne des méthodes pour le traitement ou la prévention de maladies associées à la transthyrétine (TTR-) dans lesquelles on utilise des agents d'interférence à ARN, par exemple, des agents d'interférence à ARN double brin, qui ciblent le gène de transthyrétine (TTR).
PCT/US2014/056923 2013-09-23 2014-09-23 Méthodes pour le traitement ou la prévention de maladies associées à la transthyrétine (ttr) Ceased WO2015042564A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361881257P 2013-09-23 2013-09-23
US61/881,257 2013-09-23

Publications (2)

Publication Number Publication Date
WO2015042564A1 WO2015042564A1 (fr) 2015-03-26
WO2015042564A8 true WO2015042564A8 (fr) 2015-05-28

Family

ID=51663511

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/056923 Ceased WO2015042564A1 (fr) 2013-09-23 2014-09-23 Méthodes pour le traitement ou la prévention de maladies associées à la transthyrétine (ttr)

Country Status (3)

Country Link
AR (1) AR097738A1 (fr)
TW (1) TW201610151A (fr)
WO (1) WO2015042564A1 (fr)

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EA020312B1 (ru) 2008-10-20 2014-10-30 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
US8507663B2 (en) 2010-04-06 2013-08-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of CD274/PD-L1 gene
CA2946719C (fr) 2014-03-25 2023-09-26 Arcturus Therapeutics, Inc. Oligomeres una a effets hors cible reduits pour le silencage genetique
EP3122365B1 (fr) * 2014-03-25 2023-05-03 Arcturus Therapeutics, Inc. Oligomères d'una sélectifs d'allèle de transthyrétine, pour inactivation génique
US9856475B2 (en) * 2014-03-25 2018-01-02 Arcturus Therapeutics, Inc. Formulations for treating amyloidosis
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3277289A4 (fr) * 2015-04-01 2018-12-05 Arcturus Therapeutics, Inc. Oligomères una thérapeutiques et leurs utilisations
WO2017015671A1 (fr) * 2015-07-23 2017-01-26 Arcturus Therapeutics, Inc. Compositions permettant de traiter l'amylose
BR112018000542B1 (pt) 2015-07-31 2023-01-24 Alnylam Pharmaceuticals, Inc Agente de ácido ribonucleico de fita dupla para inibição da expressão de transtiretina em uma célula, composição farmacêutica, uso dos anteriores, e método in vitro para inibição da expressão de transtiretina em uma célula
WO2017040078A1 (fr) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. Compositions d'arni ciblant le ligand de mort cellulaire programmée 1 (pd-l1) et leurs méthodes d'utilisation
MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
EP3548005A4 (fr) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes destinés à l'administration d'agents thérapeutiques
SG10201913552UA (en) * 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
NZ757927A (en) 2017-04-11 2023-07-28 Arbutus Biopharma Corp Targeted compositions
MA50267A (fr) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals Inc Compositions et méthodes de traitement de l'amylose médiée par la transthyrétine (ttr)
WO2020069055A1 (fr) * 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Compositions d'arni de la transthyrétine (ttr) et leurs procédés d'utilisation pour traiter ou prévenir des maladies oculaires associées à la ttr
CN113226332B (zh) * 2018-11-02 2025-05-09 阿布特斯生物制药公司 二价靶向缀合物
EP4055165A1 (fr) * 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Composition d'arni de la transthyrétine (ttr) et ses procédés d'utilisation pour le traitement ou la prévention de maladies oculaires associées à ttr
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
MX2024001194A (es) * 2021-08-03 2024-02-27 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de transtiretina (ttr) y sus metodos de uso.
CN119265190A (zh) * 2023-07-06 2025-01-07 上海交通大学 一种靶向TTR的saCas9 sgRNA及其修饰方式
WO2025098117A1 (fr) * 2023-11-06 2025-05-15 成都倍特药业股份有限公司 Réactif d'interférence arn, composition pour inhiber l'expression de transthyrétine (ttr) et utilisation
WO2025128622A1 (fr) * 2023-12-12 2025-06-19 Alnylam Pharmaceuticals, Inc. Méthodes et compositions pour le traitement de maladies associées à la transthyrétine (ttr)

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Also Published As

Publication number Publication date
AR097738A1 (es) 2016-04-13
WO2015042564A1 (fr) 2015-03-26
TW201610151A (zh) 2016-03-16

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