CO2021003446A2 - Agentes de arni para inhibir la expresión de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y métodos de uso - Google Patents

Agentes de arni para inhibir la expresión de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y métodos de uso

Info

Publication number
CO2021003446A2
CO2021003446A2 CONC2021/0003446A CO2021003446A CO2021003446A2 CO 2021003446 A2 CO2021003446 A2 CO 2021003446A2 CO 2021003446 A CO2021003446 A CO 2021003446A CO 2021003446 A2 CO2021003446 A2 CO 2021003446A2
Authority
CO
Colombia
Prior art keywords
hsd17b13
rnai agents
agents
methods
expression
Prior art date
Application number
CONC2021/0003446A
Other languages
English (en)
Inventor
Zhen Li
Rui Zhu
Shawn A Morales
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 CO2021003446A2 publication Critical patent/CO2021003446A2/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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/353—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente divulgación se refiere a agentes de ARNi, por ejemplo, agentes de ARNi de doble hebra, capaces de inhibir la expresión génica de 17β-hidroxiesteroide deshidrogenasa tipo 13 (HSD17B13 o 17β-HSD13). Asimismo, se dan a conocer composiciones farmacéuticas que comprenden agentes de ARNi contra HSD17B13 y métodos para la utilización de las mismas. Los agentes de ARNi contra HSD17B13 conforme a lo divulgado en la presente memoria pueden estar conjugados a ligandos dirigidos a la diana para facilitar el transporte hacia las células, incluidos los hepatocitos. El transporte de los agentes de ARNi contra HSD17B13 in vivo da lugar a la inhibición de la expresión del gen HSD17B13. Los agentes de ARNi pueden utilizarse en métodos para el tratamiento de las enfermedades y trastornos asociados con HSD17B13, incluidas la enfermedad del hígado graso no alcohólico (NAFLD), la esteatohepatitis no alcohólica (NASH), la fibrosis hepática, y las enfermedades hepáticas relacionadas o no relacionadas con el consumo de alcohol, incluida la cirrosis.
CONC2021/0003446A 2018-09-19 2021-03-17 Agentes de arni para inhibir la expresión de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y métodos de uso CO2021003446A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862733320P 2018-09-19 2018-09-19
US201862773707P 2018-11-30 2018-11-30
US201962890220P 2019-08-22 2019-08-22
PCT/US2019/051707 WO2020061177A1 (en) 2018-09-19 2019-09-18 Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
CO2021003446A2 true CO2021003446A2 (es) 2021-04-08

Family

ID=69887755

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0003446A CO2021003446A2 (es) 2018-09-19 2021-03-17 Agentes de arni para inhibir la expresión de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y métodos de uso

Country Status (20)

Country Link
US (1) US20220056454A1 (es)
EP (1) EP3852769A4 (es)
JP (1) JP7488254B2 (es)
KR (1) KR20210061380A (es)
CN (1) CN113164509A (es)
AU (1) AU2019342117A1 (es)
BR (1) BR112021005137A2 (es)
CA (1) CA3109553A1 (es)
CL (1) CL2021000669A1 (es)
CO (1) CO2021003446A2 (es)
CR (1) CR20210186A (es)
EC (1) ECSP21027262A (es)
IL (1) IL281597A (es)
MX (1) MX2021003077A (es)
PE (1) PE20211212A1 (es)
PH (1) PH12021550423A1 (es)
SG (1) SG11202101698WA (es)
TW (1) TW202024324A (es)
UY (1) UY38382A (es)
WO (1) WO2020061177A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230115344A (ko) * 2016-09-02 2023-08-02 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
SG11202007583SA (en) 2018-03-21 2020-09-29 Regeneron Pharma 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
PE20211755A1 (es) 2018-12-21 2021-09-07 Ionis Pharmaceuticals Inc Moduladores de la expresion de hsd17b13
IL298697A (en) * 2020-06-01 2023-02-01 Amgen Inc RNA interference constructs to inhibit hsd17b13 expression and methods for using them
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
JP2024502282A (ja) 2020-12-23 2024-01-18 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 細胞死誘導ｄｆｆａ様エフェクターｂ（ｃｉｄｅｂ）阻害剤での肝疾患の治療
AU2022261027A1 (en) * 2021-04-22 2023-10-26 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting 17β-hydroxysteroid dehydrogenase type 13 and sirna conjugate
CA3230382A1 (en) * 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
WO2023109935A1 (zh) * 2021-12-16 2023-06-22 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
WO2023122531A2 (en) 2021-12-20 2023-06-29 Regeneron Pharmaceuticals, Inc. Methods of identifying and evaluating liver inflammation and liver fibrosis in a subject by determining a stratified score based on gene expression
WO2023138663A1 (zh) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 一种dsRNA、其应用及制备方法
WO2023196941A1 (en) 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
US20230416268A1 (en) * 2022-04-28 2023-12-28 Enanta Pharmaceuticals, Inc. Pyrimidinone-containing 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors
CN116515835A (zh) * 2022-04-29 2023-08-01 北京福元医药股份有限公司 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途
WO2023213284A1 (zh) * 2022-05-06 2023-11-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU1067697A (en) * 1995-12-05 1997-06-27 Jouko Antero Oikarinen Hsd17b1 promoter, enhancer, silencer and use thereof
EP1752536A4 (en) * 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
CN103520724B (zh) * 2013-10-23 2016-05-25 江苏美迪森生物医药有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
JP2017535552A (ja) * 2014-11-17 2017-11-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
KR20230048446A (ko) * 2016-03-07 2023-04-11 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
UY37565A (es) * 2017-01-10 2018-07-31 Arrowhead Pharmaceuticals Inc Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso
CA3049980A1 (en) * 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
SG11202007583SA (en) * 2018-03-21 2020-09-29 Regeneron Pharma 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Also Published As

Publication number Publication date
PE20211212A1 (es) 2021-07-05
CN113164509A (zh) 2021-07-23
JP2022501040A (ja) 2022-01-06
CA3109553A1 (en) 2020-03-26
SG11202101698WA (en) 2021-04-29
CR20210186A (es) 2021-06-14
AU2019342117A1 (en) 2021-03-04
PH12021550423A1 (en) 2021-09-20
AU2019342117A2 (en) 2021-03-11
BR112021005137A2 (pt) 2021-06-15
US20220056454A1 (en) 2022-02-24
JP7488254B2 (ja) 2024-05-21
IL281597A (en) 2021-05-31
UY38382A (es) 2020-03-31
CL2021000669A1 (es) 2021-10-29
WO2020061177A1 (en) 2020-03-26
TW202024324A (zh) 2020-07-01
MX2021003077A (es) 2021-05-27
EP3852769A4 (en) 2023-12-13
ECSP21027262A (es) 2021-05-31
EP3852769A1 (en) 2021-07-28
KR20210061380A (ko) 2021-05-27

Similar Documents

Publication Publication Date Title
CO2021003446A2 (es) Agentes de arni para inhibir la expresión de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y métodos de uso
ECSP20018305A (es) Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso
CL2021001195A1 (es) Agentes de iarn para infección causada por el virus de la hepatitis b. (divisional de solicitud 201900278)
BR112014010223B8 (pt) uso de uma composição compreendendo inibidores de reciclagem de ácido de bílis e forma de dosagem pediátrica
ECSP20017905A (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA APOLIPOPROTEÍNA C-III (APOC3)
JOP20180003B1 (ar) عوامل RNAi ألفا-1 مضادة للتريبسين (AAT)، وتركيبات تتضمن عوامل AAT RNAi، وطرق الاستخدام
ECSP22075322A (es) Agentes de arni para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos, y métodos de uso
WO2018140920A8 (en) Compositions and methods for inhibition of factor xii gene expression
WO2017059223A3 (en) Compositions and methods for inhibiting gene expression of lpa
BR112018008684A2 (pt) formulações de toxina botulínica injetáveis e processos de uso das mesmas com longa duração do efeito terapêutico ou cosmético
NI201700100A (es) Composiciones farmacéuticas para terapia combinada
BR112016029781A2 (pt) composições e métodos para inibir a expressão de gene de alfa-1 antitripsina
BR112014010228A2 (pt) inibidores de reciclagem de ácidos biliares para tratamento da doença de fígado e hipercolemia colestática
BR112016024020A2 (pt) composição farmacêutica
CO2022000016A2 (es) Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd)
ECSP20055797A (es) Métodos y composiciones para tratar enfermedades pulmonares crónicas
AR114739A1 (es) AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE 17b-HSD TIPO 13 (HSD17B13), COMPOSICIONES DE DICHOS AGENTES, Y MÉTODOS DE USO
MX2020003718A (es) Composiciones y metodos para el tratamiento de fibrosis.
EA202190800A1 (ru) РНКи АГЕНТЫ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ 17БЕТА-HSD ТИПА 13 (HSD17B13), ИХ КОМПОЗИЦИИ И СПОСОБЫ ПРИМЕНЕНИЯ
EA201991675A1 (ru) СРЕДСТВА ДЛЯ РНКи АЛЬФА-1-АНТИТРИПСИНА (AAT), КОМПОЗИЦИИ, СОДЕРЖАЩИЕ СРЕДСТВА ДЛЯ РНКи AAT, И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
BR112022017963A2 (pt) Composição farmacêutica para prevenção ou tratamento de esteato-hepatite não alcoólica
AR113254A1 (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA ANGIOPOYETINA TIPO 3 (ANGPTL3) Y MÉTODOS DE USO
AR110742A1 (es) AGENTES DE iARN a-1 ANTITRIPSINA (AAT), COMPOSICIONES QUE INCLUYEN AGENTES DE iARN AAT Y MÉTODOS DE USO
AR113014A1 (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA APOLIPOPROTEÍNA C-III (APOC3)
AR113454A1 (es) AGENTES iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DEL RECEPTOR DE ASIALOGLICOPROTEÍNA 1