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MX2022006364A - Tratamiento genico con apoe. - Google Patents

Tratamiento genico con apoe.

Info

Publication number
MX2022006364A
MX2022006364A MX2022006364A MX2022006364A MX2022006364A MX 2022006364 A MX2022006364 A MX 2022006364A MX 2022006364 A MX2022006364 A MX 2022006364A MX 2022006364 A MX2022006364 A MX 2022006364A MX 2022006364 A MX2022006364 A MX 2022006364A
Authority
MX
Mexico
Prior art keywords
apoe
geneic
treatment
encodes
mammalian apolipoprotein
Prior art date
Application number
MX2022006364A
Other languages
English (en)
Inventor
Ronald G Crystal
Katie Stiles
Stephen M Kaminsky
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 MX2022006364A publication Critical patent/MX2022006364A/es

Links

Classifications

    • 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
    • C12N15/63—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
    • 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
    • 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
    • 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
    • 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)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Se proporcionan un vector de tratamiento génico que comprende un casete de expresión que codifica una apolipoproteína E de mamífero que tiene un residuo distinto de arginina en al menos una de las posiciones 112, 136 o 158, pero que no es una apolipoproteína E de mamífero que tiene R112, R136 y R158 o una apolipoproteína E de mamífero que tiene C112, R136 y C158, o que codifica un anticuerpo que se fija a APOE4 o altera la fijación de APOE a proteoglicanos de heparán sulfato, y métodos para usar el vector.
MX2022006364A 2019-11-25 2020-11-25 Tratamiento genico con apoe. MX2022006364A (es)

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
MX2022006364A true MX2022006364A (es) 2022-06-22

Family

ID=73857283

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022006364A MX2022006364A (es) 2019-11-25 2020-11-25 Tratamiento genico con apoe.

Country Status (11)

Country Link
US (1) US20230044351A1 (es)
EP (1) EP4065600A1 (es)
JP (1) JP2023502515A (es)
KR (1) KR20220107243A (es)
CN (1) CN114829390A (es)
AU (1) AU2020391518B2 (es)
BR (1) BR112022010065A2 (es)
CA (1) CA3162761A1 (es)
IL (1) IL293116A (es)
MX (1) MX2022006364A (es)
WO (1) WO2021108809A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023528797A (ja) 2020-05-26 2023-07-06 トゥルーバインディング，インコーポレイテッド ガレクチン－３を遮断することにより炎症性疾患を処置する方法
EP4508214A1 (en) * 2022-04-12 2025-02-19 uniQure biopharma B.V. Nucleic acid regulation of c9orf72
EP4508213A1 (en) * 2022-04-12 2025-02-19 uniQure biopharma B.V. Nucleic acid regulation of apoe
IL317719A (en) * 2022-07-08 2025-02-01 Univ Cornell Methods and pharmaceutical compositions for the treatment and prevention of Alzheimer's disease
WO2024220726A1 (en) * 2023-04-18 2024-10-24 Cornell University Methods and apoe pharmaceutical compositions for the treatment and the prevention of alzheimers disease

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
DE69408527T2 (de) 1993-03-09 1998-06-04 Epic Therapeutics, Inc., Norwood, Mass. Makromolekulare mikropartikel und verfahren zu ihrer herstellung
US5981719A (en) 1993-03-09 1999-11-09 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
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
EP0930874A2 (en) 1996-10-09 1999-07-28 Takeda Chemical Industries, Ltd. 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
US20140050733A1 (en) 2011-02-07 2014-02-20 Dale B. Schenk Apoe immunotherapy
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
EP3808849A1 (en) * 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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
JP2022536613A (ja) * 2019-05-28 2022-08-18 ザ ジェネラル ホスピタル コーポレイション ＡｐｏＥ抗体、融合タンパク質、およびその使用

Also Published As

Publication number Publication date
AU2020391518A1 (en) 2022-07-14
BR112022010065A2 (pt) 2022-09-06
EP4065600A1 (en) 2022-10-05
AU2020391518B2 (en) 2025-02-13
CA3162761A1 (en) 2021-06-03
IL293116A (en) 2022-07-01
US20230044351A1 (en) 2023-02-09
CN114829390A (zh) 2022-07-29
JP2023502515A (ja) 2023-01-24
KR20220107243A (ko) 2022-08-02
WO2021108809A1 (en) 2021-06-03

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