DOP2019000132A - COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS. - Google Patents

COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS.

Info

Publication number
DOP2019000132A
DOP2019000132A DO2019000132A DO2019000132A DOP2019000132A DO P2019000132 A DOP2019000132 A DO P2019000132A DO 2019000132 A DO2019000132 A DO 2019000132A DO 2019000132 A DO2019000132 A DO 2019000132A DO P2019000132 A DOP2019000132 A DO P2019000132A
Authority
DO
Dominican Republic
Prior art keywords
methods
rnai
serpina1
compositions
same
Prior art date
Application number
DO2019000132A
Other languages
English (en)
Spanish (es)
Inventor
Manoharan Muthiah
Charisse Klaus
Foster Donald
G Rajeev Kallanthottathil
K Schlegal Mark
Janas Maja
R Jadhav Vasant
V Kel'in Alexander
K Nair Jayaprakash
A Maier Martin
Matsuda Shigeo
Jayaraman Muthusamy
Sehgal Alfica
Brown Christopher
Fitzgerrald Kevin
Milstein Stuart
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 DOP2019000132A publication Critical patent/DOP2019000132A/es

Links

Classifications

    • 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 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
    • C12N2310/31—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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
DO2019000132A 2016-11-23 2019-05-23 COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS. DOP2019000132A (es)

Applications Claiming Priority (4)

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

Publications (1)

Publication Number Publication Date
DOP2019000132A true DOP2019000132A (es) 2019-08-15

Family

ID=62195353

Family Applications (1)

Application Number Title Priority Date Filing Date
DO2019000132A DOP2019000132A (es) 2016-11-23 2019-05-23 COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS.

Country Status (23)

Country Link
US (3) US10995336B2 (OSRAM)
EP (1) EP3545093A1 (OSRAM)
JP (1) JP7348067B2 (OSRAM)
KR (1) KR20190082317A (OSRAM)
CN (1) CN110114463B (OSRAM)
AU (1) AU2017363604A1 (OSRAM)
BR (1) BR112019010342A2 (OSRAM)
CA (1) CA3044562A1 (OSRAM)
CL (1) CL2019001388A1 (OSRAM)
CO (1) CO2019005992A2 (OSRAM)
CR (1) CR20190294A (OSRAM)
DO (1) DOP2019000132A (OSRAM)
IL (1) IL266746A (OSRAM)
MA (1) MA46923A (OSRAM)
MX (1) MX2019005898A (OSRAM)
MY (1) MY199978A (OSRAM)
PH (1) PH12019550076A1 (OSRAM)
RU (2) RU2021126115A (OSRAM)
SG (1) SG10201913877WA (OSRAM)
TW (2) TWI788312B (OSRAM)
UY (1) UY37494A (OSRAM)
WO (1) WO2018098117A1 (OSRAM)
ZA (1) ZA202103080B (OSRAM)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX379208B (es) 2013-05-22 2025-03-10 Alnylam Pharmaceuticals Inc Composiciones de arni de serpina1 y sus metodos de uso.
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
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
TW202436621A (zh) 2017-01-10 2024-09-16 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
MX2020009812A (es) * 2018-03-21 2021-01-08 Regeneron Pharma COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS.
CA3118537A1 (en) * 2018-11-09 2020-05-14 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotides
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
WO2021006523A1 (ko) 2019-07-08 2021-01-14 주식회사 엠디헬스케어 세균 메타게놈 분석을 통한 뇌종양 진단방법
EP4007812A1 (en) * 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
CN115484932B (zh) 2020-03-04 2024-12-10 维乎医疗有限公司 用于靶向rna递送的组合物和方法
US20230416739A1 (en) * 2020-11-13 2023-12-28 The George Washington University, A Congressionally Chartered Not-For-Profit Corporation B-spectrin (sptbn1) deficiency protects mice from high-fat diet-induced liver disease and cancer development
JP2024504505A (ja) 2021-01-30 2024-01-31 イー セラピューティクス パブリック リミテッド カンパニー コンジュゲートオリゴヌクレオチド化合物、その作製方法及び使用
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
WO2023059948A1 (en) * 2021-10-08 2023-04-13 E-Therapeutics Plc Nucleic acids containing abasic nucleosides
TW202400787A (zh) * 2022-03-16 2024-01-01 美商安彼瑞可股份有限公司 改良siRNA生物可利用性之GalNAc組合物
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
CA3259147A1 (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
WO2025136994A1 (en) * 2023-12-19 2025-06-26 Sirius Therapeutics, Inc. Modifications of polynucleic acid molecules and uses thereof

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