CO2024003968A2 - Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso - Google Patents

Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso

Info

Publication number
CO2024003968A2
CO2024003968A2 CONC2024/0003968A CO2024003968A CO2024003968A2 CO 2024003968 A2 CO2024003968 A2 CO 2024003968A2 CO 2024003968 A CO2024003968 A CO 2024003968A CO 2024003968 A2 CO2024003968 A2 CO 2024003968A2
Authority
CO
Colombia
Prior art keywords
mapt
compositions
methods
microtubule
associated protein
Prior art date
Application number
CONC2024/0003968A
Other languages
English (en)
Inventor
Jonathan Edward Farley
Mark K Schlegel
James D Mcininch
Jeffrey Zuber
Adam Castoreno
Stephen Abbott
Joseph Barry
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 CO2024003968A2 publication Critical patent/CO2024003968A2/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
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/32—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
    • C12N2310/35—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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527—Other alkyl chain
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

La descripción se refiere a agentes de interferencia de ácido ribonucleico bicatenario (ARNbci) y composiciones que se dirigen a un gen de proteína tau asociada a microtúbulos (MAPT), así como métodos para inhibir la expresión de un gen MAPT y métodos para tratar sujetos que tienen una enfermedad o trastorno asociado a MAPT, p. ej., enfermedad de Alzheimer, demencia frontotemporal, parálisis supranuclear progresiva u otras tauopatías, utilizando dichos agentes de ARNbci y composiciones.
CONC2024/0003968A 2021-09-24 2024-03-27 Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso CO2024003968A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163248119P 2021-09-24 2021-09-24
US202263321573P 2022-03-18 2022-03-18
US202263403327P 2022-09-02 2022-09-02
PCT/US2022/076979 WO2023049871A2 (en) 2021-09-24 2022-09-23 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CO2024003968A2 true CO2024003968A2 (es) 2024-04-18

Family

ID=85721285

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0003968A CO2024003968A2 (es) 2021-09-24 2024-03-27 Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso

Country Status (11)

Country Link
US (1) US20240254487A1 (es)
EP (1) EP4405484A2 (es)
JP (1) JP2024536811A (es)
KR (1) KR20240067943A (es)
AU (1) AU2022348935A1 (es)
CA (1) CA3233101A1 (es)
CO (1) CO2024003968A2 (es)
IL (1) IL311564A (es)
MX (1) MX2024003519A (es)
TW (1) TW202328449A (es)
WO (1) WO2023049871A2 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023175091A2 (en) * 2022-03-16 2023-09-21 Janssen Pharmaceutica Nv MAPT siRNA AND USES THEREOF

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6783961B1 (en) * 1999-02-26 2004-08-31 Genset S.A. Expressed sequence tags and encoded human proteins
ATE391781T1 (de) * 2002-07-12 2008-04-15 Axon Neuroscience Transgenes tier welches das trunkierte alzheimer tau protein exprimiert
AU2004295616A1 (en) * 2003-12-03 2005-06-16 Medical & Biological Laboratories Co., Ltd Fluorescent protein
TWI772856B (zh) * 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
KR101876423B1 (ko) * 2016-10-10 2018-07-09 한국과학기술연구원 치매 모델 형질전환 마우스 및 이를 이용한 스크리닝 방법
KR20210018267A (ko) * 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
CN115038789A (zh) * 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
KR20230005194A (ko) * 2020-03-30 2023-01-09 알닐람 파마슈티칼스 인코포레이티드 미세소관 관련된 단백질 TAU (MAPT) iRNA 제제 조성물 및 이의 사용 방법

Also Published As

Publication number Publication date
EP4405484A2 (en) 2024-07-31
TW202328449A (zh) 2023-07-16
US20240254487A1 (en) 2024-08-01
WO2023049871A2 (en) 2023-03-30
MX2024003519A (es) 2024-04-01
IL311564A (en) 2024-05-01
JP2024536811A (ja) 2024-10-08
CA3233101A1 (en) 2023-03-30
WO2023049871A3 (en) 2023-04-27
KR20240067943A (ko) 2024-05-17
AU2022348935A1 (en) 2024-04-04

Similar Documents

Publication Publication Date Title
CL2020002401A1 (es) Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.
CO2024003968A2 (es) Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso
BR112023016299A2 (pt) Composições e métodos para inibir kras
CO2020015314A2 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso
CL2020000991A1 (es) Proteínas trispecíficas y métodos de uso.
DOP2019000132A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS.
BR112019005548A2 (pt) tratamento da doença de huntington com aav
EA201792263A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EA201790789A1 (ru) Композиции и способы, предназначенные для ингибирования экспрессии гена hao1 (оксидазы 1 гидроксикислот (гликолат-оксидазы))
BR112018014288A2 (pt) métodos e composições para o tratamento de doença neurológica
BR112022019606A2 (pt) Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas
BR112022001324A2 (pt) Composto, composição farmacêutica e método para tratamento de uma doença mediada por p38 map quinase em um indivíduo necessitando do mesmo
CO2022016196A2 (es) Composiciones de arni del factor b del complemento (cfb) y métodos de uso de las mismas
UY34701A (es) Formas sólidas de un profármaco de nucleótidos de tiofosforamidato
EA202191630A1 (ru) ХИМИЧЕСКИ МОДИФИЦИРОВАННЫЕ КОНСТРУКЦИИ ДЛЯ RNAi И ПУТИ ИХ ПРИМЕНЕНИЯ
BR112019008810A2 (pt) micrornas modificadas com 5-halouracila e seu uso no tratamento de câncer
CO2022009787A2 (es) Agentes de arni del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72), composiciones y métodos de uso de los mismos
AR120341A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
CL2022001590A1 (es) Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos
CO2022008105A2 (es) Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos de uso
BR112022021813A2 (pt) Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
CL2022000539A1 (es) Composiciones y métodos para inhibir la expresión del gen lect2
CO2023018579A2 (es) Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina (lrrk2 ) y métodos de uso de estas
AR121138A1 (es) COMPOSICIONES DE AGENTE DE iRNA DE CINASAS 2 RICAS EN REPETICIONES DE LEUCINAS (LRRK2) Y MÉTODOS DE SU USO
AR120712A1 (es) AGENTES DE ARNi DEL MARCO DE LECTURA ABIERTO 72 DEL CROMOSOMA 9 HUMANO (C9ORF72), COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS