BR112021006520A2 - ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar - Google Patents

ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar

Info

Publication number
BR112021006520A2
BR112021006520A2 BR112021006520A BR112021006520A BR112021006520A2 BR 112021006520 A2 BR112021006520 A2 BR 112021006520A2 BR 112021006520 A BR112021006520 A BR 112021006520A BR 112021006520 A BR112021006520 A BR 112021006520A BR 112021006520 A2 BR112021006520 A2 BR 112021006520A2
Authority
BR
Brazil
Prior art keywords
double
ribonucleic acid
inhibits
complementary
expression
Prior art date
Application number
BR112021006520A
Other languages
English (en)
Inventor
Tahara Kazuhiro
Motoi Sotaro
Takahashi Yoshinori
Suzuki Yuta
Original Assignee
Eisai R&D Man Co Ltd
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 Eisai R&D Man Co Ltd filed Critical Eisai R&D Man Co Ltd
Publication of BR112021006520A2 publication Critical patent/BR112021006520A2/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005Wall or coating material
    • A61K9/5015Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/10Drugs for disorders of the urinary system of the bladder
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Dispersion Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Anti-Oxidant Or Stabilizer Compositions (AREA)
  • Cosmetics (AREA)
  • Dc Digital Transmission (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar. trata-se de um ácido ribonucleico de fita dupla que compreende uma combinação de uma fita de sentido com uma fita antissenso, sendo que a dita combinação é selecionada dentre o grupo que consiste nas seq id nos: 159 e 160, seq id nos: 141 e 142, seq id nos: 143 e 144, seq id nos: 145 e 146, seq id nos: 147 e 148 e seq id nos: 153 e 154.
BR112021006520A 2018-10-26 2019-10-24 ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar BR112021006520A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2018201777 2018-10-26
US16/354,916 US10526603B1 (en) 2018-10-26 2019-03-15 Double-stranded ribonucleic acid capable of suppressing expression of complement C5
PCT/JP2019/041783 WO2020085456A1 (ja) 2018-10-26 2019-10-24 補体c5の発現を抑制する二本鎖リボ核酸

Publications (1)

Publication Number Publication Date
BR112021006520A2 true BR112021006520A2 (pt) 2021-07-06

Family

ID=69058781

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021006520A BR112021006520A2 (pt) 2018-10-26 2019-10-24 ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar

Country Status (20)

Country Link
US (1) US10526603B1 (pt)
EP (1) EP3871680B1 (pt)
JP (1) JP6725776B1 (pt)
KR (1) KR20210086608A (pt)
CN (1) CN112771163A (pt)
AR (1) AR116832A1 (pt)
AU (1) AU2019367536A1 (pt)
BR (1) BR112021006520A2 (pt)
CA (1) CA3115249A1 (pt)
CL (2) CL2021000826A1 (pt)
CO (1) CO2021004029A2 (pt)
IL (1) IL281902B (pt)
JO (1) JOP20210066A1 (pt)
MX (1) MX2021003910A (pt)
PE (1) PE20210811A1 (pt)
PH (1) PH12021550736A1 (pt)
SG (1) SG11202102881PA (pt)
TW (2) TWI718717B (pt)
WO (1) WO2020085456A1 (pt)
ZA (1) ZA202102104B (pt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6918197B2 (ja) * 2019-12-26 2021-08-11 エーザイ・アール・アンド・ディー・マネジメント株式会社 脂質複合体を含む医薬組成物及び脂質ナノ粒子を含む医薬組成物
KR20220122608A (ko) * 2019-12-26 2022-09-02 에자이 알앤드디 매니지먼트 가부시키가이샤 보체 c5의 발현을 저해하는 이중 가닥 리보핵산을 함유하는 약제학적 조성물
WO2021154941A1 (en) * 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2023245126A2 (en) * 2022-06-15 2023-12-21 Sirnaomics, Inc. Products and compositions

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102605775B1 (ko) 2013-03-14 2023-11-29 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C5 iRNA 조성물 및 그 이용 방법
TWI648067B (zh) 2014-01-09 2019-01-21 日商衛材R&D企管股份有限公司 陽離子性脂質
EP3194596A1 (en) * 2014-09-16 2017-07-26 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
TWI699354B (zh) 2014-12-26 2020-07-21 日商衛材R&D企管股份有限公司 陽離子性脂質
US10036017B2 (en) 2015-02-17 2018-07-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA
EP4365291A2 (en) * 2015-06-12 2024-05-08 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
US20190256845A1 (en) * 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
KR102358341B1 (ko) * 2016-06-24 2022-02-07 에자이 알앤드디 매니지먼트 가부시키가이샤 양이온성 지질

Also Published As

Publication number Publication date
JPWO2020085456A1 (ja) 2021-02-15
CA3115249A1 (en) 2020-04-30
ZA202102104B (en) 2022-08-31
AU2019367536A1 (en) 2021-04-29
AR116832A1 (es) 2021-06-16
TWI718717B (zh) 2021-02-11
US10526603B1 (en) 2020-01-07
CO2021004029A2 (es) 2021-04-30
JOP20210066A1 (ar) 2023-01-30
KR20210086608A (ko) 2021-07-08
EP3871680A4 (en) 2022-09-21
IL281902A (en) 2021-05-31
JP6725776B1 (ja) 2020-07-22
TW202135833A (zh) 2021-10-01
SG11202102881PA (en) 2021-04-29
CL2023001965A1 (es) 2023-12-15
PE20210811A1 (es) 2021-04-28
EP3871680A1 (en) 2021-09-01
TW202031269A (zh) 2020-09-01
CN112771163A (zh) 2021-05-07
TWI818225B (zh) 2023-10-11
PH12021550736A1 (en) 2021-10-25
MX2021003910A (es) 2021-06-04
EP3871680B1 (en) 2024-03-20
WO2020085456A1 (ja) 2020-04-30
CL2021000826A1 (es) 2021-09-10
IL281902B (en) 2022-09-01

Similar Documents

Publication Publication Date Title
BR112021006520A2 (pt) ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar
PE20211393A1 (es) COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C5 DEL COMPLEMENTO Y METODOS PARA SU USO
CO2017007335A2 (es) Supresión del gen de la huntingtina inducida por la arni
CL2020002401A1 (es) Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.
CO2020015314A2 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso
PE20210921A1 (es) Agentes arni como inhibidores de la transcripcion de la serpina 1 y composiciones que los comprenden
NZ797319A (en) Modified rnai agents
AR090869A1 (es) COMPOSICIONES DE ARNi DE SERPINC1 Y SUS METODOS DE USO
PE20090064A1 (es) Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
CO2020001354A2 (es) Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia un arni, por ejemplo, un ácido ribonucleico de hebra doble (arnhd), composiciones dirigidas al gen serpinc1 y métodos para usar dicho arni, por ejemplo, arnhd, composiciones para tratar un evento de sangrado en un sujeto que tiene una hemofilia (por ejemplo, con o sin inhibidores).
BR112016022742A2 (pt) composições e métodos para modular a expressão de receptor de hormônio do crescimento
BR112016007226A2 (pt) composições e métodos para inibição da expressão do gene alas1
TR201818825T4 (tr) Kardi̇yak hi̇pertrofi̇ni̇n tedavi̇si̇ne ve tanisina yöneli̇k lncrnalar
AR100946A1 (es) Composiciones y métodos para regular la expresión génica por medio de la interferencia de arn
CL2021001552A1 (es) Moléculas inhibidoras de ácido nucleico de doble hebra que contienen un triloop
MX2020010802A (es) Moleculas inhibidoras de acidos nucleicos de caracter bicatenario modificadas con nucleotidos que aumentan la tm.
BR112018070249A2 (pt) sistema promotor de u6 modificada para expressão específica de tecido
AR120341A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
AR083445A1 (es) siARN CONTRA LA FIBROSIS
BR112019010486A2 (pt) sirna quimicamente modificado
CO2021010304A2 (es) Composiciones de arni de serpinc1 y métodos de uso de las mismas
BR112022010742A2 (pt) Composição farmacêutica contendo ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar
ES2671745T3 (es) Detección de Neisseria gonorrhoeae
BR112015022885A2 (pt) composições de promotor
AR127877A2 (es) COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO