UY37565A - Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso - Google Patents

Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso

Info

Publication number
UY37565A
UY37565A UY0001037565A UY37565A UY37565A UY 37565 A UY37565 A UY 37565A UY 0001037565 A UY0001037565 A UY 0001037565A UY 37565 A UY37565 A UY 37565A UY 37565 A UY37565 A UY 37565A
Authority
UY
Uruguay
Prior art keywords
aat
agents
iarn
methods
compositions including
Prior art date
Application number
UY0001037565A
Other languages
English (en)
Inventor
Tao Pei
Zhen Li
Rui Zhu
I Wooddell Christine
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of UY37565A publication Critical patent/UY37565A/es

Links

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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/81—Protease inhibitors
    • G01N2333/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • G01N2333/811—Serine protease (E.C. 3.4.21) inhibitors
    • G01N2333/8121—Serpins
    • G01N2333/8125—Alpha-1-antitrypsin
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/08—Hepato-biliairy disorders other than hepatitis
    • G01N2800/085—Liver diseases, e.g. portal hypertension, fibrosis, cirrhosis, bilirubin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Se describen agentes de iARN para inhibir la expresión del gen alfa-1 antitripsina (AAT), composiciones que incluyen agente de iARN AAT y métodos para su uso, También se describen composiciones farmacéuticas que comprenden uno o más agentes de iARN AAT junto con uno o más excipientes capaces de suministrar uno o más agentes de iARN AAT a una célula hepática in vivo. El suministro de uno o más agentes de iARN AAT a células hepáticas in vivo prevé la inhibición de la expresión del gen AAT y el tratamiento de enfermedades asociadas con la deficiencia de AAT, como hepatitis crónica.
UY0001037565A 2017-01-10 2018-01-10 Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso UY37565A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762444452P 2017-01-10 2017-01-10
US201762486720P 2017-04-18 2017-04-18
US201762596232P 2017-12-08 2017-12-08

Publications (1)

Publication Number Publication Date
UY37565A true UY37565A (es) 2018-07-31

Family

ID=62782328

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001037565A UY37565A (es) 2017-01-10 2018-01-10 Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso

Country Status (14)

Country Link
US (4) US10450565B2 (es)
EP (1) EP3568482A4 (es)
JP (2) JP7258773B2 (es)
CN (1) CN110268060A (es)
AU (1) AU2018208505B2 (es)
BR (1) BR112019014282A2 (es)
CA (1) CA3045045A1 (es)
IL (2) IL310548A (es)
JO (1) JOP20180003B1 (es)
MX (1) MX2019008252A (es)
TW (1) TWI826365B (es)
UY (1) UY37565A (es)
WO (1) WO2018132432A1 (es)
ZA (2) ZA201903348B (es)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012178033A2 (en) * 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
CN113018307A (zh) 2013-05-22 2021-06-25 阿尔尼拉姆医药品有限公司 SERPINA1 iRNA组合物及其使用方法
CN113797348A (zh) * 2016-03-07 2021-12-17 箭头药业股份有限公司 用于治疗性化合物的靶向配体
EP3464313A4 (en) * 2016-06-06 2020-02-26 Arrowhead Pharmaceuticals, Inc. MODIFIED NUCLEOTIDES 5 '-CYCLO-PHOSPHONATE
AU2017320582B2 (en) * 2016-09-02 2023-11-16 Arrowhead Pharmaceuticals, Inc Targeting ligands
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
JOP20180003B1 (ar) * 2017-01-10 2022-09-15 Arrowhead Pharmaceuticals Inc عوامل RNAi ألفا-1 مضادة للتريبسين (AAT)، وتركيبات تتضمن عوامل AAT RNAi، وطرق الاستخدام
EP4219713A3 (en) 2017-04-05 2023-08-16 Silence Therapeutics GmbH Products and compositions
CN108931653A (zh) * 2018-08-31 2018-12-04 广州华澳生物科技有限公司 一种α1- 抗胰蛋白酶免疫层析检测试剂卡及其制备方法
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
JP2023501020A (ja) * 2018-12-28 2023-01-18 サーナオミクス インコーポレイテッド 治療用分子の標的化送達
TW202113081A (zh) * 2019-06-06 2021-04-01 美商愛羅海德製藥公司 用於治療α-1抗胰蛋白酶缺乏症(AATD)的方法
EP4071163A4 (en) * 2020-01-30 2024-03-20 Eisai R&D Management Co., Ltd. NUCLEIC ACID COMPLEX AND PHARMACEUTICAL COMPOSITION CONTAINING SAME
US20230348905A1 (en) 2020-09-15 2023-11-02 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
AU2022215065A1 (en) * 2021-01-30 2023-07-27 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2022162161A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2024062413A1 (en) * 2022-09-21 2024-03-28 Janssen Biotech, Inc. Novel stabilized nucleoside phosphates and analogues thereof

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
AU598946B2 (en) 1987-06-24 1990-07-05 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
JP2823959B2 (ja) 1991-10-24 1998-11-11 アイシス・ファーマシューティカルス・インコーポレーテッド 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
AU3206697A (en) 1996-05-17 1997-12-09 Thomas Jefferson University Ribozyme-mediated gene replacement
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
EP2341058A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
FR2774379B1 (fr) 1998-01-30 2002-03-29 Groupe Limagrain Holding Procede de production, par des cellules vegetales, d'alpha 1-antitrypsine et de ses variantes, et produits contenant l'alpha-antitrypsine ainsi obtenue
US6271358B1 (en) 1998-07-27 2001-08-07 Isis Pharmaceuticals, Inc. RNA targeted 2′-modified oligonucleotides that are conformationally preorganized
NZ513402A (en) 1999-02-12 2003-06-30 Sankyo Co Novel nucleosides and oligonucleotide analogues
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
US8137695B2 (en) 2006-08-18 2012-03-20 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
AU2001249622B2 (en) 2000-03-30 2007-06-07 Massachusetts Institute Of Technology RNA sequence-specific mediators of RNA interference
HU230458B1 (hu) 2000-12-01 2016-07-28 Europäisches Laboratorium für Molekularbiologie (EMBL) Az RNS interferenciát közvetítő kis RNS molekulák
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
EP2141234B1 (en) 2003-03-21 2016-04-27 Roche Innovation Center Copenhagen A/S Short Interfering RNA (siRNA) Analogues
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US8084599B2 (en) 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
DK1809302T3 (da) 2004-10-20 2012-11-26 Antisense Therapeutics Ltd Antisense-modulation af integrin-alfa-4-ekspression
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US20100048680A1 (en) 2007-01-11 2010-02-25 University Of Florida Research Foundation Inc. Alpha-1-Antitrypsin Variants and Uses Thereof
MX2009012568A (es) 2007-05-22 2009-12-08 Mdrna Inc Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico.
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
CA2718765A1 (en) 2008-03-20 2009-09-24 Quark Pharmaceuticals, Inc. Novel sirna compounds for inhibiting rtp801
AU2009336191B2 (en) 2008-12-18 2017-08-24 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
PL2539451T3 (pl) 2010-02-24 2016-08-31 Arrowhead Res Corporation Kompozycje do ukierunkowanego dostarczania siRNA
WO2012033848A1 (en) 2010-09-07 2012-03-15 Integrated Dna Technologies, Inc. Modifications for antisense compounds
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
WO2012178033A2 (en) * 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
CA2842039A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
AU2012377385A1 (en) 2012-04-18 2014-01-23 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
CN113018307A (zh) * 2013-05-22 2021-06-25 阿尔尼拉姆医药品有限公司 SERPINA1 iRNA组合物及其使用方法
WO2014197524A2 (en) 2013-06-06 2014-12-11 Serpin Pharma, Llc Peptide therapy for treatment of alpha-1 antitrypsin deficiency and associated pathologies
JP2016528887A (ja) 2013-07-03 2016-09-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物
US20170114341A1 (en) * 2014-06-06 2017-04-27 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
KR20180038465A (ko) * 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
WO2017139616A1 (en) 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
CN113797348A (zh) 2016-03-07 2021-12-17 箭头药业股份有限公司 用于治疗性化合物的靶向配体
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
AU2017320582B2 (en) 2016-09-02 2023-11-16 Arrowhead Pharmaceuticals, Inc Targeting ligands
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
JOP20180003B1 (ar) 2017-01-10 2022-09-15 Arrowhead Pharmaceuticals Inc عوامل RNAi ألفا-1 مضادة للتريبسين (AAT)، وتركيبات تتضمن عوامل AAT RNAi، وطرق الاستخدام
SE540888C2 (en) 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
TW202113081A (zh) 2019-06-06 2021-04-01 美商愛羅海德製藥公司 用於治療α-1抗胰蛋白酶缺乏症(AATD)的方法

Also Published As

Publication number Publication date
US20220170015A1 (en) 2022-06-02
EP3568482A1 (en) 2019-11-20
TW201831686A (zh) 2018-09-01
ZA202002652B (en) 2022-12-21
AU2018208505A1 (en) 2019-06-20
IL310548A (en) 2024-03-01
CN110268060A (zh) 2019-09-20
JOP20180003A1 (ar) 2019-01-30
AU2018208505B2 (en) 2024-03-07
US11203756B2 (en) 2021-12-21
MX2019008252A (es) 2019-09-06
IL267959A (en) 2019-09-26
ZA201903348B (en) 2020-09-30
US10450565B2 (en) 2019-10-22
BR112019014282A2 (pt) 2020-03-03
WO2018132432A8 (en) 2019-07-04
US20200208149A1 (en) 2020-07-02
US20240141351A1 (en) 2024-05-02
KR20190098748A (ko) 2019-08-22
WO2018132432A1 (en) 2018-07-19
JP2020503070A (ja) 2020-01-30
CA3045045A1 (en) 2018-07-19
JOP20180003B1 (ar) 2022-09-15
JP7258773B2 (ja) 2023-04-17
IL267959B1 (en) 2024-03-01
EP3568482A4 (en) 2020-12-16
US11884920B2 (en) 2024-01-30
TWI826365B (zh) 2023-12-21
US20180195069A1 (en) 2018-07-12
JP2023076634A (ja) 2023-06-01

Similar Documents

Publication Publication Date Title
UY37565A (es) Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso
CO2018003678A2 (es) Composiciones y métodos para inhibir la expresión génica de lpa
MX2023003672A (es) Composiciones y metodos para inhibir la expresion del gen de alfa-1 antitripsina.
DOP2019000005A (es) 3-metil pirazinas 2,5-disustituidas y 3-metil pirazinas 2,5,6-trisustituidas como inhibidores alostéricos de shp2
ECSP20025070A (es) Proteínas trispecificas y métodos de uso
MX2017011422A (es) Composiciones y metodos para inhibir la expresion del gen del factor xii.
DOP2015000108A (es) Compuestos de pirrolopirimidina como inhibidores de quinasas
MX2020010412A (es) Inhibidores de tirosina quinasa de bruton.
GT201500020A (es) Sulfamoilarilamidas y su uso como medicamentos para el tratamiento de la hepatitis b
CL2015000829A1 (es) Inhibidores de histona desmetilasas
BR112016029041A8 (pt) uso de um inibidor de glutaminase, composição farmacêutica e kit farmacêutico
BR112017015310A2 (pt) uso de pgr4 como um agente anti-inflamatório
ECSP15004752A (es) Formulaciones de etanercept que muestran una marcada reducción de la cantidad de partículas subvisibles
TW201613919A (en) Inhibitors of Bruton's tyrosine kinase
UY35703A (es) Anticuerpos contra el inhibidor-1 del activador de plasminógeno (pai-1) y usos de los mismos
CO6280488A2 (es) Combinacion farmaceutica
CL2020000019A1 (es) Agentes de iarn para la inhibición de la expresión de alfa-enac y métodos de uso.
AR107935A1 (es) Uso del compuesto 6-(4-(2,3-dimetilfenoxi)piperidin-1-il)metil)pirimidino-2,4(1h,3h)-diona o una sal farmacéuticamente aceptable del mismo para preparar un medicamento, combinación y composición farmacéutica que comprende dicho compuesto
AR110742A1 (es) AGENTES DE iARN a-1 ANTITRIPSINA (AAT), COMPOSICIONES QUE INCLUYEN AGENTES DE iARN AAT Y MÉTODOS DE USO
UY38937A (es) AGENTES DE iARN PARA INHIBIR LA EXPRESIÓN DEL ENaC BETA, COMPOSICIONES DE ESTOS Y MÉTODOS DE USO
EA201991675A1 (ru) СРЕДСТВА ДЛЯ РНКи АЛЬФА-1-АНТИТРИПСИНА (AAT), КОМПОЗИЦИИ, СОДЕРЖАЩИЕ СРЕДСТВА ДЛЯ РНКи AAT, И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
MX2019000717A (es) Composiciones que comprenden una matriz de polisacarido para la liberacion controlada de ingredientes activos.
AR112754A1 (es) AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE a-ENaC Y MÉTODOS DE USO
AR106227A1 (es) COMPOSICIONES Y MÉTODOS PARA INHIBIR LA EXPRESIÓN GÉNICA DEL GEN DE LA APOLIPOPROTEÍNA APO(a) (LPA)
LV15451A (lv) Līdzeklis mitohondriālās DNS daudzuma paaugstināšanai zīdītāju šūnās

Legal Events

Date Code Title Description
109 Application deemed to be withdrawn

Effective date: 20231026