WO2024056902A3 - Compositions et méthodes de traitement de maladies neurologiques - Google Patents

Compositions et méthodes de traitement de maladies neurologiques Download PDF

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Publication number
WO2024056902A3
WO2024056902A3 PCT/EP2023/075536 EP2023075536W WO2024056902A3 WO 2024056902 A3 WO2024056902 A3 WO 2024056902A3 EP 2023075536 W EP2023075536 W EP 2023075536W WO 2024056902 A3 WO2024056902 A3 WO 2024056902A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
compositions
neurological diseases
atxn2
expression
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/EP2023/075536
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English (en)
Other versions
WO2024056902A2 (fr
Inventor
Christopher Shaw
Youn Bok Lee
Romain JOUBERT
Do Young Lee
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.)
Individual
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Individual
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Publication date
Application filed by Individual filed Critical Individual
Priority to EP23773208.6A priority Critical patent/EP4587568A2/fr
Priority to JP2025515736A priority patent/JP2025531201A/ja
Priority to CA3267488A priority patent/CA3267488A1/fr
Priority to AU2023342734A priority patent/AU2023342734A1/en
Publication of WO2024056902A2 publication Critical patent/WO2024056902A2/fr
Publication of WO2024056902A3 publication Critical patent/WO2024056902A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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
    • 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

Landscapes

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

Abstract

L'invention concerne des compositions et des méthodes pour le traitement de troubles associés à l'expression de transcrits d'ARN d'ataxine-2 (ATXN2) de type sauvage ou mutants, comprenant des troubles caractérisés par des gènes contenant des régions de répétition étendues de manière aberrante qui peuvent conduire à un phénotype pathologique. L'invention concerne des constructions d'ARN inhibitrices qui suppriment l'expression d'ATXN2, ainsi que des vecteurs viraux, tels que des vecteurs viraux adéno-associés, codant pour de telles molécules d'ARN inhibitrices.
PCT/EP2023/075536 2022-09-16 2023-09-15 Compositions et méthodes de traitement de maladies neurologiques Ceased WO2024056902A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP23773208.6A EP4587568A2 (fr) 2022-09-16 2023-09-15 Compositions et méthodes de traitement de maladies neurologiques
JP2025515736A JP2025531201A (ja) 2022-09-16 2023-09-15 神経疾患を治療するための組成物及び方法
CA3267488A CA3267488A1 (fr) 2022-09-16 2023-09-15 Compositions et méthodes de traitement de maladies neurologiques
AU2023342734A AU2023342734A1 (en) 2022-09-16 2023-09-15 Compositions and methods for treating neurological diseases

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263407321P 2022-09-16 2022-09-16
US63/407,321 2022-09-16

Publications (2)

Publication Number Publication Date
WO2024056902A2 WO2024056902A2 (fr) 2024-03-21
WO2024056902A3 true WO2024056902A3 (fr) 2024-04-18

Family

ID=88143828

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2023/075536 Ceased WO2024056902A2 (fr) 2022-09-16 2023-09-15 Compositions et méthodes de traitement de maladies neurologiques

Country Status (5)

Country Link
EP (1) EP4587568A2 (fr)
JP (1) JP2025531201A (fr)
AU (1) AU2023342734A1 (fr)
CA (1) CA3267488A1 (fr)
WO (1) WO2024056902A2 (fr)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080113351A1 (en) * 2004-05-11 2008-05-15 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
WO2020023737A1 (fr) * 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Composés et méthodes permettant de réduire l'expression d'atxn2
WO2021159008A2 (fr) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions et méthodes de traitement de maladies neurodégénératives
WO2022026531A1 (fr) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Compositions d'arni atxn2 et leurs procédés d'utilisation pour traiter ou prévenir des maladies neurodégénératives associées à atxn2

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5766960A (en) 1987-07-27 1998-06-16 Australian Membrane And Biotechnology Research Institute Receptor membranes
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DK0752248T3 (da) 1992-11-13 2000-11-13 Idec Pharma Corp Terapeutisk anvendelse af kimæriske og radioaktivt mærkede antistoffer mod humant B-lymfocytbegrænset differentieringsantig
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6268210B1 (en) 1998-05-27 2001-07-31 Hyseq, Inc. Sandwich arrays of biological compounds
US6232068B1 (en) 1999-01-22 2001-05-15 Rosetta Inpharmatics, Inc. Monitoring of gene expression by detecting hybridization to nucleic acid arrays using anti-heteronucleic acid antibodies
DE10120829A1 (de) 2000-04-27 2001-12-20 Max Delbrueck Centrum Sleeping Beauty, ein Transposonvektor mit breitem Wirtsbereich für die genetische Transformation bei Wirbeltieren
JP5170934B2 (ja) 2001-08-16 2013-03-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 改良ｄｎａリポフェクションならびに／または徐放性プロドラッグおよび薬物療法のための試薬の合成と使用
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
US20080313773A1 (en) 2007-05-14 2008-12-18 The Rockefeller University Production of artificial micrornas using synthetic microrna precursors
EP1995316A1 (fr) 2007-05-25 2008-11-26 Qiagen GmbH Procédé de purification de cellules préservant lesdites cellules, obtention de cellules et transfection de cellules
EP2009095A1 (fr) 2007-06-28 2008-12-31 Innovalor AG Procédé de génération de céllules sensibles au glucose
EP2215223B1 (fr) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Méganucléases monocaténaires conçues rationnellement contenant des séquences de reconnaissance non palindromiques
WO2010085699A2 (fr) 2009-01-23 2010-07-29 The Johns Hopkins University Transposon piggybac de mammifère et procédés d'utilisation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080113351A1 (en) * 2004-05-11 2008-05-15 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
WO2020023737A1 (fr) * 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Composés et méthodes permettant de réduire l'expression d'atxn2
WO2021159008A2 (fr) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions et méthodes de traitement de maladies neurodégénératives
WO2022026531A1 (fr) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Compositions d'arni atxn2 et leurs procédés d'utilisation pour traiter ou prévenir des maladies neurodégénératives associées à atxn2

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LINDSAY A. BECKER ET AL: "Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice", NATURE, vol. 544, no. 7650, 1 April 2017 (2017-04-01), pages 367 - 371, XP055607892, DOI: 10.1038/nature22038 *

Also Published As

Publication number Publication date
AU2023342734A1 (en) 2025-03-27
WO2024056902A2 (fr) 2024-03-21
EP4587568A2 (fr) 2025-07-23
JP2025531201A (ja) 2025-09-19
CA3267488A1 (fr) 2024-03-21

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