AR124302A1 - Gápmeros de oligonucleótidos de objetivo tau - Google Patents

Gápmeros de oligonucleótidos de objetivo tau

Info

Publication number
AR124302A1
AR124302A1 ARP210103430A ARP210103430A AR124302A1 AR 124302 A1 AR124302 A1 AR 124302A1 AR P210103430 A ARP210103430 A AR P210103430A AR P210103430 A ARP210103430 A AR P210103430A AR 124302 A1 AR124302 A1 AR 124302A1
Authority
AR
Argentina
Prior art keywords
tau
gapmers
targeting oligonucleotide
seq
inhibition
Prior art date
Application number
ARP210103430A
Other languages
English (en)
Inventor
Dae Shik Kim
Hyeong Wook Choi
Francis G Fang
Yoshinori Takahashi
Kenji Kikuta
Hikaru YOSHIMURA
Wataru Itano
Toshiki Kurokawa
Ryo Dairiki
Zhi Zhou
Mingde Shan
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 AR124302A1 publication Critical patent/AR124302A1/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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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

Landscapes

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

Abstract

Se proporcionan oligonucleótidos antisentido. Estos oligonucleótidos antisentido son útiles en la preparación de gápmeros para la inhibición de la transcripción del ARNm de Tau. La inhibición de la transcripción del ARNm de Tau puede dar como resultado la disminución de las cantidades de proteína Tau en un sujeto, lo que permite el tratamiento de enfermedades y trastornos relacionados con la expresión de Tau, incluyendo la enfermedad de Alzheimer y las taupatías primarias. Reivindicación 1: Un gápmero oligonucleotídico antisentido o una sal farmacéuticamente aceptable del mismo que comprende una secuencia de nucleótidos seleccionada del grupo que consiste en la SEQ ID Nº 1 hasta la SEQ ID Nº 17.
ARP210103430A 2020-12-11 2021-12-10 Gápmeros de oligonucleótidos de objetivo tau AR124302A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063124447P 2020-12-11 2020-12-11

Publications (1)

Publication Number Publication Date
AR124302A1 true AR124302A1 (es) 2023-03-15

Family

ID=79287725

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210103430A AR124302A1 (es) 2020-12-11 2021-12-10 Gápmeros de oligonucleótidos de objetivo tau

Country Status (4)

Country Link
US (1) US20220195437A1 (es)
AR (1) AR124302A1 (es)
TW (1) TW202237848A (es)
WO (1) WO2022125984A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
CN119816508A (zh) * 2022-07-07 2025-04-11 卫材R&D管理有限公司 用于制备反义寡核苷酸的结晶单体及其制备方法和用途
KR20250039397A (ko) * 2022-07-15 2025-03-20 엔트라다 테라퓨틱스, 인크. 하이브리드 올리고뉴클레오티드

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
UY36550A (es) * 2015-02-04 2016-08-31 Roche Innovation Ct Copenhagen As Oligómeros antisentido de tau y sus usos
WO2017024264A2 (en) 2015-08-05 2017-02-09 Eisai R&D Management Co., Ltd. Chiral reagents for preparation of homogeneous oligomers
CN109937042B (zh) 2016-09-20 2023-06-27 科罗拉多州立大学董事会法人团体 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体
JOP20190065A1 (ar) * 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau

Also Published As

Publication number Publication date
TW202237848A (zh) 2022-10-01
WO2022125984A1 (en) 2022-06-16
US20220195437A1 (en) 2022-06-23

Similar Documents

Publication Publication Date Title
AR124302A1 (es) Gápmeros de oligonucleótidos de objetivo tau
ES2749374T3 (es) Compuestos inhibitorios dirigidos a conexina 43 y sus métodos de uso en el tratamiento del trauma ocular de la córnea
US9840707B2 (en) Modified TGF-beta2 oligonucleotides
KR102825946B1 (ko) IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
ES2325660T3 (es) Oligomeros nucleobasicos de iap no codificantes y usos de los mismos.
JP2015518711A (ja) Ｂｄｎｆ発現を調節するための組成物及び方法
JP2016531570A (ja) ユークロマチン領域を標的とするオリゴヌクレオチド
JP2015518713A (ja) Ｕｔｒｎ発現を調節するための組成物及び方法
JP2015519057A (ja) Ｐｔｅｎ発現を調節するための組成物及び方法
JP2015523853A (ja) Ａｔｐ２ａ２発現を調節するための組成物及び方法
JP2015518710A (ja) ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
MX2025008039A (es) Uso de oligonucleótidos antisentido multisegmentados modificados
MXPA06004920A (es) Tratamiento de enfermedades proliferativas usando un olig??mero antisentido de iap y un agente quimioterapeutico.
JP2016528258A (ja) 非コードｒｎａを形成するヘテロクロマチン
PE20211749A1 (es) Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
US10066230B2 (en) Immune regulatory oligonucleotide (IRO) compounds to modulate toll-like receptor based immune response
CN110234764A (zh) 同时抑制mTOR基因及STAT3基因表达的核酸
AR125992A1 (es) Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)
PE20252304A1 (es) Oligonucleotidos antisentido y su uso para el tratamiento de trastornos neurodegenerativos
US20030119776A1 (en) Modulation of Fas and FasL expression
CN104995300B (zh) Rna活性和血管通透性的调节
KR20190128806A (ko) 암의 예방 또는 치료용 약학적 조성물
KR20180028690A (ko) Linc-ASEN 발현 억제제를 유효성분으로 포함하는 세포 노화 유도용 조성물
KR101783444B1 (ko) miR-33-5p 를 이용한 뇌신경세포 보호 물질 스크리닝 방법
WO2023129939A2 (en) Anti-sense oligonucleotides and uses thereof

Legal Events

Date Code Title Description
FB Suspension of granting procedure