AR102777A1 - COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO - Google Patents

COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO

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Publication number
AR102777A1
AR102777A1 ARP150103845A ARP150103845A AR102777A1 AR 102777 A1 AR102777 A1 AR 102777A1 AR P150103845 A ARP150103845 A AR P150103845A AR P150103845 A ARP150103845 A AR P150103845A AR 102777 A1 AR102777 A1 AR 102777A1
Authority
AR
Argentina
Prior art keywords
methods
tmprss6
rnai agents
arni
compositions
Prior art date
Application number
ARP150103845A
Other languages
English (en)
Inventor
Milstein Stuart
Jadhav Vasant
Foster Donald
Fishman Shannon
Fitzgerald Kevin
Maier Martin
Butler James
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 AR102777A1 publication Critical patent/AR102777A1/es

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    • 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
    • C12N15/1137—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 enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • Epidemiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La solicitud se relaciona con agentes de ARNi, por ejemplo, agentes de ARNi de doble hebra, que se dirigen contra el gen Proteasa Transmembrana, Serina 6 (TMPRSS6), y métodos para usar dichos agentes de ARNi para inhibir la expresión de TMPRSS6 y métodos para tratar sujetos que tienen un trastorno asociado con TMPRSS6, por ejemplo, un trastorno asociado a sobrecarga de hierro, tal como b-talasemia o hemocromatosis. Reivindicación 2: El agente de ARNi de doble hebra de la reivindicación 1, donde hebra sentido y la hebra antisentido comprenden las secuencias nucleotídicas de hebra sentido y antisentido de un dúplex que se elige del grupo que consiste en AD-63202, AD-64372, AD-64373, AD-64374, AD-64375, AD-64376, AD-64377, AD-64378, AD-64380, AD-64381, AD-64382, AD-64384, AD-64385, AD-64386, AD-64387, AD-64389, AD-64601, AD-64569, AD-64604, AD-64567, AD-60940, AD-64601, AD-65105, AD-65106, AD-65107, AD-65108, AD-65109, AD-65110, AD-65111, AD-65112, AD-61002, AD-66014, AD-66015 y AD-65189.
ARP150103845A 2014-11-24 2015-11-24 COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO AR102777A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462083691P 2014-11-24 2014-11-24

Publications (1)

Publication Number Publication Date
AR102777A1 true AR102777A1 (es) 2017-03-22

Family

ID=55022672

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150103845A AR102777A1 (es) 2014-11-24 2015-11-24 COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO

Country Status (4)

Country Link
US (3) US10246713B2 (es)
AR (1) AR102777A1 (es)
TW (1) TW201629218A (es)
WO (1) WO2016085852A1 (es)

Families Citing this family (15)

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KR102365961B1 (ko) 2011-03-29 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
KR102486617B1 (ko) 2013-05-22 2023-01-12 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
WO2016085852A1 (en) 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
WO2018185240A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
UY37803A (es) * 2017-07-10 2019-02-28 Genzyme Corp Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia
EP3598995A1 (en) * 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
CA3118537A1 (en) 2018-11-09 2020-05-14 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotides
WO2021105389A1 (en) * 2019-11-29 2021-06-03 Kymab Limited Treatment for physiological iron overload
MX2022009763A (es) * 2020-02-10 2022-09-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion del factor de crecimiento endotelial vascular a (vegf-a).
MX2023012586A (es) * 2021-04-26 2023-10-31 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de serina 6, proteasa transmembranaria (tmprss6) y sus metodos de uso.
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
EP4687983A1 (en) 2023-03-27 2026-02-11 Silence Therapeutics GmbH Compounds and compositions for use in stem cell transplantation
WO2025148896A1 (en) * 2024-01-08 2025-07-17 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6)

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Also Published As

Publication number Publication date
US20190284562A1 (en) 2019-09-19
WO2016085852A1 (en) 2016-06-02
US11725209B2 (en) 2023-08-15
US10246713B2 (en) 2019-04-02
US20210163952A1 (en) 2021-06-03
US20170362594A1 (en) 2017-12-21
TW201629218A (zh) 2016-08-16
US10829763B2 (en) 2020-11-10

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