BR112019010342A2 - composições do irna de serpina1 e métodos de uso destas - Google Patents

composições do irna de serpina1 e métodos de uso destas

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Publication number
BR112019010342A2
BR112019010342A2 BR112019010342A BR112019010342A BR112019010342A2 BR 112019010342 A2 BR112019010342 A2 BR 112019010342A2 BR 112019010342 A BR112019010342 A BR 112019010342A BR 112019010342 A BR112019010342 A BR 112019010342A BR 112019010342 A2 BR112019010342 A2 BR 112019010342A2
Authority
BR
Brazil
Prior art keywords
methods
serpina1
irna compositions
serpina1 irna
agents
Prior art date
Application number
BR112019010342A
Other languages
English (en)
Inventor
V Kel'in Alexander
Sehgal Alfica
Brown Christopher
Foster Donald
K Nair Jayaprakash
G Rajeev Kallanthottathil
Fitzgerald Kevin
Charisse Klaus
Janas Maja
K Schlegal Mark
A Maier Martin
Manoharan Muthiah
Jayaraman Muthusamy
Matsuda Shigeo
Milstein Stuart
R Jadhav Vasant
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 BR112019010342A2 publication Critical patent/BR112019010342A2/pt

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

a presente invenção refere-se a agentes rnai, por exemplo, agentes rnai fita dupla, direcionados para o gene serpina1, e métodos de utilização de tais agentes rnai para inibir a expressão de serpina1 e métodos para o tratamento de indivíduos com uma doença associada à serpina1, como um transtorno hepático.
BR112019010342A 2016-11-23 2017-11-21 composições do irna de serpina1 e métodos de uso destas BR112019010342A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662425907P 2016-11-23 2016-11-23
US201762548589P 2017-08-22 2017-08-22
US201762549099P 2017-08-23 2017-08-23
US201762561514P 2017-09-21 2017-09-21
PCT/US2017/062701 WO2018098117A1 (en) 2016-11-23 2017-11-21 SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
BR112019010342A2 true BR112019010342A2 (pt) 2019-10-22

Family

ID=62195353

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019010342A BR112019010342A2 (pt) 2016-11-23 2017-11-21 composições do irna de serpina1 e métodos de uso destas

Country Status (22)

Country Link
US (2) US10995336B2 (pt)
EP (1) EP3545093A1 (pt)
JP (1) JP7348067B2 (pt)
KR (1) KR20190082317A (pt)
CN (1) CN110114463B (pt)
AU (1) AU2017363604A1 (pt)
BR (1) BR112019010342A2 (pt)
CA (1) CA3044562A1 (pt)
CL (1) CL2019001388A1 (pt)
CO (1) CO2019005992A2 (pt)
CR (1) CR20190294A (pt)
DO (1) DOP2019000132A (pt)
IL (1) IL266746A (pt)
MA (1) MA46923A (pt)
MX (1) MX2019005898A (pt)
PH (1) PH12019550076A1 (pt)
RU (2) RU2761667C2 (pt)
SG (1) SG10201913877WA (pt)
TW (2) TW202313978A (pt)
UY (1) UY37494A (pt)
WO (1) WO2018098117A1 (pt)
ZA (1) ZA202103080B (pt)

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SI2999785T1 (en) 2013-05-22 2018-08-31 Alnylam Pharmaceuticals, Inc. SERPINA1 IRNA assemblies and procedures for their use
TW202313978A (zh) 2016-11-23 2023-04-01 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
IL267959B2 (en) 2017-01-10 2024-07-01 Arrowhead Pharmaceuticals Inc Alpha-1 antitrypsin (AAT) RNAi agents, preparations containing AAT RNAi agents and their uses
AU2019239971A1 (en) * 2018-03-21 2020-09-03 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
JP7536007B2 (ja) * 2018-11-09 2024-08-19 アルニラム ファーマスーティカルズ インコーポレイテッド 修飾二重鎖オリゴヌクレオチド
JP2022532652A (ja) * 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの経口送達
JP7304565B2 (ja) 2019-07-08 2023-07-07 エムディー ヘルスケア インコーポレイテッド 細菌メタゲノム解析を通した脳腫瘍診断方法
GB202411424D0 (en) 2020-03-04 2024-09-18 Verve Therapeutics Inc Compositions and methods for targeted rna delivery
JP2023549505A (ja) * 2020-11-13 2023-11-27 ザ ジョージ ワシントン ユニバーシティ， ア コングレッショナリー チャータード ノット－フォー－プロフィット コーポレイション Ｂ－スペクトリン（ｓｐｔｂｎ１）欠損はマウスを高脂肪食起因性肝疾患及び癌への進展から保護する
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
EP4413130A1 (en) * 2021-10-08 2024-08-14 E-Therapeutics plc Nucleic acids containing abasic nucleosides
US11879125B2 (en) * 2022-03-16 2024-01-23 Empirico Inc. GalNAc compositions for improving siRNA bioavailability
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
CN117070583B (zh) * 2023-10-16 2024-05-14 吉林凯莱英制药有限公司 抑制PCSK9基因表达的siRNA的制备方法

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