AR127942A1 - Agentes de interferencia de arn de mapt - Google Patents

Agentes de interferencia de arn de mapt

Info

Publication number
AR127942A1
AR127942A1 ARP220103406A ARP220103406A AR127942A1 AR 127942 A1 AR127942 A1 AR 127942A1 AR P220103406 A ARP220103406 A AR P220103406A AR P220103406 A ARP220103406 A AR P220103406A AR 127942 A1 AR127942 A1 AR 127942A1
Authority
AR
Argentina
Prior art keywords
mapt
rna interference
interference agents
agents
mapt rna
Prior art date
Application number
ARP220103406A
Other languages
English (en)
Inventor
Sarah Katharina Fritschi
Valcarcel Isabel Cristina Gonzalez
Andrew Peter Mcarthy
Rebecca Ruth Miles
Douglas Raymond Perkins
Keith Geoffrey Phillips
Kaushambi Roy
Jibo Wang
Shih Wu
- York Jeremy S Ying
Barbara Calamani
Original Assignee
Lilly Co Eli
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 Lilly Co Eli filed Critical Lilly Co Eli
Publication of AR127942A1 publication Critical patent/AR127942A1/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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

En la presente descripción se proporcionan agentes de ARNi de MAPT y composiciones que comprenden un agente de ARNi de MAPT. En la presente descripción también se proporcionan métodos para usar los agentes de ARNi de MAPT o composiciones que comprenden un agente de ARNi de MAPT para reducir la expresión de MAPT y/o tratar una tauopatía en un sujeto.
ARP220103406A 2021-12-13 2022-12-13 Agentes de interferencia de arn de mapt AR127942A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163288846P 2021-12-13 2021-12-13

Publications (1)

Publication Number Publication Date
AR127942A1 true AR127942A1 (es) 2024-03-13

Family

ID=85036477

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220103406A AR127942A1 (es) 2021-12-13 2022-12-13 Agentes de interferencia de arn de mapt

Country Status (4)

Country Link
US (2) US20230212576A1 (es)
AR (1) AR127942A1 (es)
TW (1) TW202342748A (es)
WO (1) WO2023114700A1 (es)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US10513701B2 (en) * 2015-03-25 2019-12-24 Università Degli Studi Di Trento RNA interference mediated therapy for neurodegenerative diseases
JP7353301B2 (ja) 2018-05-07 2023-09-29 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 肝臓外送達
WO2020188626A1 (ja) 2019-03-15 2020-09-24 和夫 金子 視覚支援装置
CN115552006A (zh) * 2020-03-18 2022-12-30 马萨诸塞大学 用于mapt调节的寡核苷酸
CN116234585A (zh) * 2020-03-30 2023-06-06 阿尔尼拉姆医药品有限公司 微管相关蛋白TAU(MAPT)iRNA剂组合物及其使用方法
WO2023064707A1 (en) 2021-10-07 2023-04-20 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing tau expression

Also Published As

Publication number Publication date
WO2023114700A1 (en) 2023-06-22
US20230212576A1 (en) 2023-07-06
TW202342748A (zh) 2023-11-01
US20230340482A1 (en) 2023-10-26
US11926827B2 (en) 2024-03-12

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