WO2024201423A3 - Arnsi ciblant la protéine tau associée aux microtubules et leurs utilisations - Google Patents

Arnsi ciblant la protéine tau associée aux microtubules et leurs utilisations Download PDF

Info

Publication number
WO2024201423A3
WO2024201423A3 PCT/IB2024/053120 IB2024053120W WO2024201423A3 WO 2024201423 A3 WO2024201423 A3 WO 2024201423A3 IB 2024053120 W IB2024053120 W IB 2024053120W WO 2024201423 A3 WO2024201423 A3 WO 2024201423A3
Authority
WO
WIPO (PCT)
Prior art keywords
microtubule
associated protein
protein tau
targeting sirnas
mapt
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/IB2024/053120
Other languages
English (en)
Other versions
WO2024201423A2 (fr
Inventor
Jin-Hyeob RYU
Young Jin Park
Suhyeong Park
Yu Na Lim
Insun Kim
Mincheol Kwon
Jihye Kim
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biorchestra Co Ltd
Original Assignee
Biorchestra 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 Biorchestra Co Ltd filed Critical Biorchestra Co Ltd
Publication of WO2024201423A2 publication Critical patent/WO2024201423A2/fr
Publication of WO2024201423A3 publication Critical patent/WO2024201423A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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 nucleic acids [NA]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)
  • Epidemiology (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)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne un ARNsi qui cible un transcrit MAPT dans une cellule, conduisant à une expression réduite de la protéine MAPT. La réduction de l'expression de la protéine MAPT est bénéfique pour le traitement de certains troubles médicaux, par exemple, une tauopathie.
PCT/IB2024/053120 2023-03-30 2024-03-29 Arnsi ciblant la protéine tau associée aux microtubules et leurs utilisations Ceased WO2024201423A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363493244P 2023-03-30 2023-03-30
US63/493,244 2023-03-30

Publications (2)

Publication Number Publication Date
WO2024201423A2 WO2024201423A2 (fr) 2024-10-03
WO2024201423A3 true WO2024201423A3 (fr) 2024-12-05

Family

ID=92903490

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2024/053120 Ceased WO2024201423A2 (fr) 2023-03-30 2024-03-29 Arnsi ciblant la protéine tau associée aux microtubules et leurs utilisations

Country Status (1)

Country Link
WO (1) WO2024201423A2 (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016151523A1 (fr) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Traitement à médiation par l'interférence arn des maladies neurodégénératives
WO2021202511A2 (fr) * 2020-03-30 2021-10-07 Alnylam Pharmaceuticals, Inc. Compositions d'agent d'arni à protéine tau associée aux microtubules (mapt) et leurs procédés d'utilisation
US20210363523A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for mapt modulation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016151523A1 (fr) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Traitement à médiation par l'interférence arn des maladies neurodégénératives
US20210363523A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for mapt modulation
WO2021202511A2 (fr) * 2020-03-30 2021-10-07 Alnylam Pharmaceuticals, Inc. Compositions d'agent d'arni à protéine tau associée aux microtubules (mapt) et leurs procédés d'utilisation

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
3 March 2023 (2023-03-03), ANONYMOUS: "PREDICTED: Desmodus rotundus microtubule associated protein tau (MAPT), transcript variant X7, mRNA", XP009559235 *
4 March 2023 (2023-03-04), ANONYMOUS: "PREDICTED: Sciurus carolinensis microtubule associated protein tau (Mapt), transcript variant X4, mRNA", XP009559233 *

Also Published As

Publication number Publication date
WO2024201423A2 (fr) 2024-10-03

Similar Documents

Publication Publication Date Title
WO2022032154A3 (fr) Compositions pour l'administration de molécules de charge utile à l'épithélium des voies respiratoires
CR20200301A (es) Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos
MX2020008581A (es) Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
CN100577680C (zh) 对Syk激酶表达的抑制
WO2022272248A9 (fr) Modulateurs de cbl-b et leurs utilisations
WO2011049414A3 (fr) Procédé pour l'induction d'une migration de cellules souches adultes dérivées de tissu adipeux
WO2007087113A3 (fr) Utilisation de transcrits d'arn antisens et non codants naturels comme cibles de médicaments
WO2008156702A3 (fr) Extinction de gènes médiée par des bactéries
WO2010096170A3 (fr) Inhibition d'arnt synthétases et ses applications thérapeutiques
WO2004039957A3 (fr) Inhibition de l'expression de genes au moyen d'agents d'interference a arn
WO2024201423A3 (fr) Arnsi ciblant la protéine tau associée aux microtubules et leurs utilisations
Wang et al. Echinococcus granulosus sensu stricto: silencing of thioredoxin peroxidase impairs the differentiation of protoscoleces into metacestodes
CN104388225A (zh) 一种养生葡萄酒及其制备方法
WO2021231771A3 (fr) Traitement du cancer en utilisant un arnsi pour moduler l'expression de la protéine prdm2/riz
Tong et al. MiR-146a negatively regulates Aspergillus fumigatus-induced TNF-α and IL-6 secretion in THP-1 macrophages
Lee et al. A glycosidic spinasterol from Koreana stewartia promotes procollagen production and inhibits matrix metalloproteinase-1 expression in UVB-irradiated human dermal fibroblasts
WO2006102684A3 (fr) Administration ex vivo, dans des cellules, de genes, de proteines et de medicaments mediee par un reseau de champ electrique de force ultrafaible (lsefn)
WO2024086692A3 (fr) Expression d'un gène ou d'une protéine induite par un virus de l'herpès et méthodes de traitement de troubles neurologiques
Shin et al. Effect of eosinophils activated with Alternaria on the production of extracellular matrix from nasal fibroblasts
WO2020206115A3 (fr) Oligonucléotides antisens angptl2 et leurs utilisations
WO2023197001A3 (fr) Compositions et méthodes de traitement de maladies hépatiques avec des arnsi ciblant cideb
WO2022067340A3 (fr) Méthodes et compositions de traitement de maladies neurodégénératives
Baryshok et al. 1-Germatranol hydrate as an activator of tryptophanyl-tRNA synthetase
WO2024151942A3 (fr) Arnsi ciblant des transcrits slc10a1, compositions et utilisations associées
CN102209536B (zh) 用于抑制表皮增生和减轻炎性皮肤疾病的含邻二羟基异黄酮衍生物的皮肤外用组合物

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 24778445

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 24778445

Country of ref document: EP

Kind code of ref document: A2