JP2025517123A - アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子 - Google Patents

アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子 Download PDF

Info

Publication number
JP2025517123A
JP2025517123A JP2024564846A JP2024564846A JP2025517123A JP 2025517123 A JP2025517123 A JP 2025517123A JP 2024564846 A JP2024564846 A JP 2024564846A JP 2024564846 A JP2024564846 A JP 2024564846A JP 2025517123 A JP2025517123 A JP 2025517123A
Authority
JP
Japan
Prior art keywords
mapt
hnrnp
tau
mrna
axonal
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.)
Pending
Application number
JP2024564846A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023217437A5 (https=
JP2025517123A5 (https=
Inventor
アントン セントナー，ミハエル
ブリース，ミハエル
ザレ，アブドルホセイン
サリーシ，サエデ
Original Assignee
ユリウス－マクシミリアンズ－ウニヴェルジテート ウュルツブルグ
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 ユリウス－マクシミリアンズ－ウニヴェルジテート ウュルツブルグ filed Critical ユリウス－マクシミリアンズ－ウニヴェルジテート ウュルツブルグ
Publication of JP2025517123A publication Critical patent/JP2025517123A/ja
Publication of JPWO2023217437A5 publication Critical patent/JPWO2023217437A5/ja
Publication of JP2025517123A5 publication Critical patent/JP2025517123A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024564846A 2022-05-13 2023-03-13 アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子 Pending JP2025517123A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263364629P 2022-05-13 2022-05-13
US63/364,629 2022-05-13
US202263382536P 2022-11-07 2022-11-07
US63/382,536 2022-11-07
PCT/EP2023/056392 WO2023217437A1 (en) 2022-05-13 2023-03-13 Method and molecules for reducing axonal tau protein accumulation through blocking of hnrnp r-mediated mapt mrna transport for treatment of alzheimer's disease

Publications (3)

Publication Number Publication Date
JP2025517123A true JP2025517123A (ja) 2025-06-03
JPWO2023217437A5 JPWO2023217437A5 (https=) 2025-12-09
JP2025517123A5 JP2025517123A5 (https=) 2025-12-09

Family

ID=85685250

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024564846A Pending JP2025517123A (ja) 2022-05-13 2023-03-13 アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子

Country Status (6)

Country Link
US (1) US20250304958A1 (https=)
EP (1) EP4522217A1 (https=)
JP (1) JP2025517123A (https=)
AU (1) AU2023267136A1 (https=)
CA (1) CA3246379A1 (https=)
WO (1) WO2023217437A1 (https=)

Family Cites Families (3)

* 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
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
AU2021238319A1 (en) * 2020-03-18 2022-10-06 University Of Massachusetts Oligonucleotides for MAPT modulation

Also Published As

Publication number Publication date
CA3246379A1 (en) 2023-11-16
WO2023217437A1 (en) 2023-11-16
EP4522217A1 (en) 2025-03-19
AU2023267136A1 (en) 2024-10-10
US20250304958A1 (en) 2025-10-02

Similar Documents

Publication Publication Date Title
Long et al. Novel upregulation of amyloid-β precursor protein (APP) by microRNA-346 via targeting of APP mRNA 5′-untranslated region: Implications in Alzheimer’s disease
Litvinchuk et al. Apolipoprotein E4 reduction with antisense oligonucleotides decreases neurodegeneration in a tauopathy model
Zanotti et al. Exosomes and exosomal miRNAs from muscle-derived fibroblasts promote skeletal muscle fibrosis
Chen et al. Parkinson’s disease-related Leucine-rich repeat kinase 2 modulates nuclear morphology and genomic stability in striatal projection neurons during aging
Liufu et al. The polyG diseases: a new disease entity
EP3362564B1 (en) Nucleic acid based tia-1 inhibitors
JP2019500428A (ja) タウ発現を減少させるための組成物および方法
JP2018531046A6 (ja) 核酸ベースのｔｉａ−１阻害剤
JP2023525799A (ja) 状態及び疾患の治療のためのｏｐａ１アンチセンスオリゴマー
US20250059535A1 (en) Opa1 antisense oligomers for treatment of conditions and diseases
WO2019229133A1 (en) Methods of treating or preventing an aging-related disease
Weiss et al. The nuclear matrix protein Matr3 regulates processing of the synaptic microRNA-138-5p
US20240352461A1 (en) Restoration of the cftr function by splicing modulation
Haify et al. Small molecule 1a reduces FMRpolyG-mediated toxicity in in vitro and in vivo models for FMR1 premutation
Januel et al. Moxifloxacin rescues SMA phenotypes in patient-derived cells and animal model
US12409189B2 (en) STAUFEN1 agents and associated methods
JP2023549160A (ja) 神経変性疾患の治療のために神経細胞におけるｃｈｍｐ７発現を阻害するための方法
US12584133B2 (en) Antisense nucleic acid and use thereof
JP2025517123A (ja) アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子
Konar et al. Damage-induced senescent immune cells regulate regeneration of the zebrafish retina
US12565650B2 (en) STAUFEN1 regulating agents and associated methods
Ahammad et al. Anti-Sense Oligonucleotide as a Therapeutic for Synucleinopathies: Pharmacokinetic, Safety and Efficacy Evaluation
Sun et al. Significant downregulation of Alzheimer's amyloid-β levels enabled by engineered DNA nanomaterials
Carlini et al. Identification of p38 MAPK inhibition as a neuroprotective strategy for combinatorial SMA therapy
Ahammad et al. Peptide-Mediated Delivery of α-Synuclein Targeting ASO: Pharmacokinetics, Safety, and CNS Efficacy in a Synucleinopathy Model

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251128

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251128