AU2020391518B2 - APOE gene therapy - Google Patents

APOE gene therapy Download PDF

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Publication number
AU2020391518B2
AU2020391518B2 AU2020391518A AU2020391518A AU2020391518B2 AU 2020391518 B2 AU2020391518 B2 AU 2020391518B2 AU 2020391518 A AU2020391518 A AU 2020391518A AU 2020391518 A AU2020391518 A AU 2020391518A AU 2020391518 B2 AU2020391518 B2 AU 2020391518B2
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AU
Australia
Prior art keywords
apoe2
vector
apoe
apoe4
aav
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AU2020391518A
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English (en)
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AU2020391518A1 (en
Inventor
Ronald G. Crystal
Stephen M. Kaminsky
Dolan Sondhi
Katie STILES
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Cornell University
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Cornell University
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Publication of AU2020391518A1 publication Critical patent/AU2020391518A1/en
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Publication of AU2020391518B2 publication Critical patent/AU2020391518B2/en
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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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • 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]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2020391518A 2019-11-25 2020-11-25 APOE gene therapy Active AU2020391518B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962939999P 2019-11-25 2019-11-25
US62/939,999 2019-11-25
PCT/US2020/070822 WO2021108809A1 (en) 2019-11-25 2020-11-25 Apoe gene therapy

Publications (2)

Publication Number Publication Date
AU2020391518A1 AU2020391518A1 (en) 2022-07-14
AU2020391518B2 true AU2020391518B2 (en) 2025-02-13

Family

ID=73857283

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020391518A Active AU2020391518B2 (en) 2019-11-25 2020-11-25 APOE gene therapy

Country Status (11)

Country Link
EP (1) EP4065600A1 (https=)
JP (2) JP2023502515A (https=)
KR (1) KR20220107243A (https=)
CN (1) CN114829390A (https=)
AU (1) AU2020391518B2 (https=)
BR (1) BR112022010065A2 (https=)
CA (1) CA3162761A1 (https=)
IL (1) IL293116A (https=)
MX (1) MX2022006364A (https=)
NZ (1) NZ789753A (https=)
WO (1) WO2021108809A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY204234A (en) 2018-08-27 2024-08-16 Regeneron Pharma Use of raman spectroscopy in downstream purification
AU2020214796A1 (en) 2019-01-30 2021-07-29 Truebinding, Inc. Anti-Gal3 antibodies and uses thereof
WO2021242776A2 (en) 2020-05-26 2021-12-02 Truebinding, Inc. Methods of treating inflammatory diseases by blocking galectin-3
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
EP4508213A1 (en) * 2022-04-12 2025-02-19 uniQure biopharma B.V. Nucleic acid regulation of apoe
WO2023198702A1 (en) * 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
EP4551711A1 (en) * 2022-07-08 2025-05-14 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease
WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019028306A2 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES

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AU4198793A (en) 1992-07-24 1994-01-27 Takeda Chemical Industries Ltd. Microparticle preparation and production thereof
FR2695563B1 (fr) 1992-09-11 1994-12-02 Pasteur Institut Microparticules portant des antigènes et leur utilisation pour l'induction de réponses humorales ou cellulaires.
US6090925A (en) 1993-03-09 2000-07-18 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
US5981719A (en) 1993-03-09 1999-11-09 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
ATE163230T1 (de) 1993-03-09 1998-02-15 Epic Therapeutics Inc Makromolekulare mikropartikel und verfahren zu ihrer herstellung
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EP2673635A4 (en) 2011-02-07 2015-08-12 Neotope Biosciences Ltd IMMUNOTHERAPY THROUGH APOE
WO2012115806A1 (en) 2011-02-24 2012-08-30 University Of Iowa Research Foundation New biodegradable polymers with sulfenamide bonds for drug delivery applications
CN106890315B (zh) * 2017-03-16 2019-09-06 北京热休生物技术有限公司 Apo-e蛋白及其多肽在治疗与预防癌症中的应用
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy

Also Published As

Publication number Publication date
NZ789753A (en) 2026-02-27
CA3162761A1 (en) 2021-06-03
MX2022006364A (es) 2022-06-22
KR20220107243A (ko) 2022-08-02
WO2021108809A1 (en) 2021-06-03
US20230044351A1 (en) 2023-02-09
CN114829390A (zh) 2022-07-29
JP2023502515A (ja) 2023-01-24
BR112022010065A2 (pt) 2022-09-06
JP2025163078A (ja) 2025-10-28
EP4065600A1 (en) 2022-10-05
AU2020391518A1 (en) 2022-07-14
IL293116A (en) 2022-07-01

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