NZ789753A - Apoe gene therapy - Google Patents

Apoe gene therapy

Info

Publication number
NZ789753A
NZ789753A NZ789753A NZ78975320A NZ789753A NZ 789753 A NZ789753 A NZ 789753A NZ 789753 A NZ789753 A NZ 789753A NZ 78975320 A NZ78975320 A NZ 78975320A NZ 789753 A NZ789753 A NZ 789753A
Authority
NZ
New Zealand
Prior art keywords
gene therapy
apoe gene
coding
mammalian apolipoprotein
apoe
Prior art date
Application number
NZ789753A
Other languages
English (en)
Inventor
Ronald G Crystal
Stephen M Kaminsky
Katie Stiles
Dolan Sondhi
Original Assignee
Univ Cornell
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 Univ Cornell filed Critical Univ Cornell
Publication of NZ789753A publication Critical patent/NZ789753A/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775Apolipopeptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
NZ789753A 2019-11-25 2020-11-25 Apoe gene therapy NZ789753A (en)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
NZ789753A true NZ789753A (en) 2026-02-27

Family

ID=73857283

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ789753A NZ789753A (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=)

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

Family Cites Families (24)

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US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
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
US5603960A (en) 1993-05-25 1997-02-18 O'hagan; Derek T. Preparation of microparticles and method of immunization
US6913767B1 (en) 1993-10-25 2005-07-05 Genentech, Inc. Compositions for microencapsulation of antigens for use as vaccines
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
CA2267930A1 (en) 1996-10-09 1998-04-16 Nobuyuki Takechi A method for producing a microparticle
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
US6884435B1 (en) 1997-01-30 2005-04-26 Chiron Corporation Microparticles with adsorbent surfaces, methods of making same, and uses thereof
US6406719B1 (en) 1998-05-13 2002-06-18 Microbiological Research Authority Encapsulation of bioactive agents
GB9810236D0 (en) 1998-05-13 1998-07-08 Microbiological Res Authority Improvements relating to encapsulation of bioactive agents
US6264987B1 (en) 2000-05-19 2001-07-24 Alkermes Controlled Therapeutics Inc. Ii Method for preparing microparticles having a selected polymer molecular weight
WO2007041190A2 (en) 2005-09-30 2007-04-12 The University Of Iowa Research Foundation Polymer-based delivery system for immunotherapy of cancer
US9572894B2 (en) 2010-10-18 2017-02-21 The University Of Iowa Research Foundation Biodegradable particulate formulations
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
JP7221275B2 (ja) * 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
CN109776665B (zh) * 2019-02-02 2021-02-05 首都医科大学宣武医院 阿尔茨海默病新突变、其稳转细胞模型及医药用途
CA3141988A1 (en) * 2019-05-28 2020-12-03 The General Hospital Corporation Apoe antibodies, fusion proteins and uses thereof

Also Published As

Publication number Publication date
AU2020391518B2 (en) 2025-02-13
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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