MA46923A - Compositions à base d'arni ciblant le gène serpina1 et leurs méthodes d'utilisation - Google Patents

Compositions à base d'arni ciblant le gène serpina1 et leurs méthodes d'utilisation

Info

Publication number
MA46923A
MA46923A MA046923A MA46923A MA46923A MA 46923 A MA46923 A MA 46923A MA 046923 A MA046923 A MA 046923A MA 46923 A MA46923 A MA 46923A MA 46923 A MA46923 A MA 46923A
Authority
MA
Morocco
Prior art keywords
rnai
methods
based compositions
serpina1 gene
compositions targeting
Prior art date
Application number
MA046923A
Other languages
English (en)
Inventor
Christopher Brown
Klaus Charisse
Kevin Fitzgerrald
Donald Foster
Vasant R Jadhav
Maja Janas
Muthusamy Jayaraman
Alexander V Kel'in
Martin A Maier
Muthiah Manoharan
Shigeo Matsuda
Stuart Milstein
Jayaprakash K Nair
Kallanthottathil G Rajeev
Mark K Schlegal
Alfica Sehgal
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 MA46923A publication Critical patent/MA46923A/fr

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
    • 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
    • C12N15/11—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
    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MA046923A 2016-11-23 2017-11-21 Compositions à base d'arni ciblant le gène serpina1 et leurs méthodes d'utilisation MA46923A (fr)

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
MA46923A true MA46923A (fr) 2019-10-02

Family

ID=62195353

Family Applications (1)

Application Number Title Priority Date Filing Date
MA046923A MA46923A (fr) 2016-11-23 2017-11-21 Compositions à base d'arni ciblant le gène serpina1 et leurs méthodes d'utilisation

Country Status (22)

Country Link
US (2) US10995336B2 (fr)
EP (1) EP3545093A1 (fr)
JP (1) JP7348067B2 (fr)
KR (1) KR20190082317A (fr)
CN (1) CN110114463A (fr)
AU (1) AU2017363604A1 (fr)
BR (1) BR112019010342A2 (fr)
CA (1) CA3044562A1 (fr)
CL (1) CL2019001388A1 (fr)
CO (1) CO2019005992A2 (fr)
CR (1) CR20190294A (fr)
DO (1) DOP2019000132A (fr)
IL (1) IL266746A (fr)
MA (1) MA46923A (fr)
MX (1) MX2019005898A (fr)
PH (1) PH12019550076A1 (fr)
RU (2) RU2021126115A (fr)
SG (1) SG10201913877WA (fr)
TW (2) TWI788312B (fr)
UY (1) UY37494A (fr)
WO (1) WO2018098117A1 (fr)
ZA (1) ZA202103080B (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN110268060A (zh) 2017-01-10 2019-09-20 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
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.
JP2022506503A (ja) * 2018-11-09 2022-01-17 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
CA3138915A1 (fr) * 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Administration orale d'oligonucleotides
WO2021006523A1 (fr) 2019-07-08 2021-01-14 주식회사 엠디헬스케어 Méthode de diagnostic d'une tumeur cérébrale au moyen d'une analyse métagénomique bactérienne
WO2021178725A1 (fr) 2020-03-04 2021-09-10 Verve Therapeutics, Inc. Compositions et méthodes d'administration ciblée d'arn
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
EP4176061A1 (fr) * 2021-01-30 2023-05-10 E-Therapeutics plc Acides nucléiques contenant des nucléotides abasiques
WO2023059948A1 (fr) * 2021-10-08 2023-04-13 E-Therapeutics Plc Acides nucléiques contenant des nucléosides abasiques
WO2023178144A2 (fr) * 2022-03-16 2023-09-21 Empirico Inc. Compositions de galnac pour améliorer la biodisponibilité de l'arnsi
WO2024023256A1 (fr) * 2022-07-27 2024-02-01 E-Therapeutics Plc Composés d'acide nucléique
CN117070583A (zh) * 2023-10-16 2023-11-17 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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