AU2021344607A1 - Methods for treating neurological disease - Google Patents

Methods for treating neurological disease Download PDF

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Publication number
AU2021344607A1
AU2021344607A1 AU2021344607A AU2021344607A AU2021344607A1 AU 2021344607 A1 AU2021344607 A1 AU 2021344607A1 AU 2021344607 A AU2021344607 A AU 2021344607A AU 2021344607 A AU2021344607 A AU 2021344607A AU 2021344607 A1 AU2021344607 A1 AU 2021344607A1
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AU
Australia
Prior art keywords
disease
composition
vector
nucleic acid
sequence
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AU2021344607A
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English (en)
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AU2021344607A9 (en
Inventor
Anne BRAAE
Philippe Moullier
Michael L. Roberts
Lester SUAREZ
Anna Tretiakova
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AskBio Inc
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Asklepios Biopharmaceutical Inc
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Publication of AU2021344607A1 publication Critical patent/AU2021344607A1/en
Publication of AU2021344607A9 publication Critical patent/AU2021344607A9/en
Assigned to AskBio Inc. reassignment AskBio Inc. Amend patent request/document other than specification (104) Assignors: ASKLEPIOS BIOPHARMACEUTICAL, INC.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0073—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen 1.14.13
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/14—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced flavin or flavoprotein as one donor, and incorporation of one atom of oxygen (1.14.14)
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13098—Cholesterol 24-hydroxylase (1.14.13.98)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2021344607A 2020-09-21 2021-09-21 Methods for treating neurological disease Pending AU2021344607A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063080925P 2020-09-21 2020-09-21
US63/080,925 2020-09-21
US202063121152P 2020-12-03 2020-12-03
US63/121,152 2020-12-03
US202163139410P 2021-01-20 2021-01-20
US63/139,410 2021-01-20
US202163140440P 2021-01-22 2021-01-22
US63/140,440 2021-01-22
US202163180407P 2021-04-27 2021-04-27
US63/180,407 2021-04-27
PCT/US2021/071534 WO2022061378A2 (en) 2020-09-21 2021-09-21 Methods for treating neurological disease

Publications (2)

Publication Number Publication Date
AU2021344607A1 true AU2021344607A1 (en) 2023-06-01
AU2021344607A9 AU2021344607A9 (en) 2024-09-12

Family

ID=80776430

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021344607A Pending AU2021344607A1 (en) 2020-09-21 2021-09-21 Methods for treating neurological disease

Country Status (7)

Country Link
US (1) US20230365963A1 (https=)
EP (1) EP4213891A4 (https=)
JP (1) JP2023542211A (https=)
AU (1) AU2021344607A1 (https=)
CA (1) CA3193406A1 (https=)
IL (1) IL301525A (https=)
WO (1) WO2022061378A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
JP2025503910A (ja) * 2022-01-27 2025-02-06 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 神経疾患を処置するための組成物
WO2023212521A2 (en) * 2022-04-25 2023-11-02 Myrtelle Inc. Treatments of disorders of myelin
EP4601743A1 (en) * 2022-10-14 2025-08-20 Institut National de la Santé et de la Recherche Médicale Methods and pharmaceutical composition for the treatment of alpha-synucleinopathies
WO2024149844A1 (en) * 2023-01-12 2024-07-18 Nantes Université Chemically-modified adeno-associated virus

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003288231A1 (en) * 2002-12-13 2004-07-09 Bayer Healthcare Ag Cholesterol 24-hydroxylase (cyp46) as therapeutic target for the treatment of alzheimer's disease
EP2187898B8 (en) * 2007-09-12 2016-09-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of viral vectors carrying the cyp46a1 gene for the treatment of alzheimer's disease
US8900627B2 (en) 2008-06-06 2014-12-02 Mirna Therapeutics, Inc. Compositions for the in vivo delivery of RNAi agents
US9132173B2 (en) * 2010-10-15 2015-09-15 Institut National De La Sante Et De La Recherche Medicale (Inserm) Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease
KR101367568B1 (ko) * 2011-05-31 2014-02-27 임우석 Ａｂｃ 수송체 조절을 통한 퇴행성신경질환에서 이상단백질 조절
BR112014006596A2 (pt) 2011-09-21 2017-03-28 Yissum Res Dev Co nanopartícula que encapsula uma pluralidade de nanocarreadores, composição, uso de uma nanopartícula, kit, sistema de entrega, processo para obter uma nanopartícula, e, processo de nanoaspersão para obter as nanopartículas
ES2661236T3 (es) 2011-12-13 2018-03-28 Henry Ford Health System Procedimientos, sistemas y composiciones para administración de microARN derivado de células/a base de vesículas
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
KR20180063954A (ko) * 2016-12-02 2018-06-14 서울대학교산학협력단 알츠하이머병 치료를 위한 마이크로RNA-188-5p의 용도
WO2019177550A1 (en) 2018-03-10 2019-09-19 Koc Universitesi Therapeutic nanoparticles containing argonaute for microrna delivery and compositions and methods using same
US20200071699A1 (en) * 2018-08-31 2020-03-05 Ghassan S. Kassab Treatment of alzheimers disease with micro rna and ghrelin
US20220054597A1 (en) * 2018-10-29 2022-02-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Expression vector for cholesterol 24-hydrolase in therapy of amyotrophic lateral sclerosis

Also Published As

Publication number Publication date
US20230365963A1 (en) 2023-11-16
CA3193406A1 (en) 2022-03-24
WO2022061378A2 (en) 2022-03-24
IL301525A (en) 2023-05-01
JP2023542211A (ja) 2023-10-05
EP4213891A4 (en) 2025-07-23
WO2022061378A3 (en) 2022-04-28
EP4213891A2 (en) 2023-07-26
AU2021344607A9 (en) 2024-09-12

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