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WO2018129203A3 - Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination - Google Patents

Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination Download PDF

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Publication number
WO2018129203A3
WO2018129203A3 PCT/US2018/012409 US2018012409W WO2018129203A3 WO 2018129203 A3 WO2018129203 A3 WO 2018129203A3 US 2018012409 W US2018012409 W US 2018012409W WO 2018129203 A3 WO2018129203 A3 WO 2018129203A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
tissue
temporal
drug delivery
specific drug
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/US2018/012409
Other languages
French (fr)
Other versions
WO2018129203A2 (en
Inventor
Cheng-Han Chen
Benjamin M. Wu
Reza Ardehali
Ngoc Bao Nguyen
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.)
University of California Berkeley
University of California San Diego UCSD
Original Assignee
University of California Berkeley
University of California San Diego UCSD
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 University of California Berkeley, University of California San Diego UCSD filed Critical University of California Berkeley
Publication of WO2018129203A2 publication Critical patent/WO2018129203A2/en
Publication of WO2018129203A3 publication Critical patent/WO2018129203A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A61K47/6935—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol
    • A61K47/6937—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol the polymer being PLGA, PLA or polyglycolic acid
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/203—Animal model comprising inducible/conditional expression system, e.g. hormones, tet
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Epidemiology (AREA)
  • Nanotechnology (AREA)
  • Animal Husbandry (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

In certain embodiments METHODS for conditional and target-specific recombination are provided where the methods comprise: providing a mammal comprising cells that express an ER-ligand-inducible CRE recombinase under the control of a promoter, and that comprises a nucleic acid sequence flanked by a pair of loxP sequences; and administering to the mammal biodegradable polymer nanoparticles containing an estrogen receptor ligand where the nanoparticles provide specific delivery to a target tissue of the estrogen receptor ligand thereby activating said CRE recombinase which performs target specific recombination of the nucleic acid between the loxP sequences in said target tissue.
PCT/US2018/012409 2017-01-06 2018-01-04 Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination Ceased WO2018129203A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762443604P 2017-01-06 2017-01-06
US62/443,604 2017-01-06

Publications (2)

Publication Number Publication Date
WO2018129203A2 WO2018129203A2 (en) 2018-07-12
WO2018129203A3 true WO2018129203A3 (en) 2018-08-16

Family

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Family Applications (1)

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PCT/US2018/012409 Ceased WO2018129203A2 (en) 2017-01-06 2018-01-04 Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination

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WO (1) WO2018129203A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
WO2020102382A1 (en) * 2018-11-13 2020-05-22 H. Lee Moffitt Cancer Center And Research Institute Inc. Tlr9-targeted therapeutics
WO2020247815A1 (en) * 2019-06-06 2020-12-10 Cornell University Transgenic rodent model for lung fibrosis and uses thereof
CN112626116B (en) * 2019-10-08 2022-11-15 黄菁 Method for site-specific integration of large-fragment exogenous DNA
CA3157333A1 (en) * 2019-10-10 2021-04-15 Solid Biosciences Inc. Modified aav capsids and uses thereof

Citations (10)

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WO2002028175A2 (en) * 2000-10-03 2002-04-11 Association Pour Le Developpement De La Recherche En Genetique Moleculaire (Aderegem) Transgenic mouse for targeted recombination mediated by modified cre-er
US20040023203A1 (en) * 2002-05-31 2004-02-05 Gero Miesenbock Heterologous stimulus-gated ion channels and methods of using same
US20050013869A1 (en) * 2003-07-18 2005-01-20 Chaw Cheng Shu Sustained release formulation for carbamates and a method therefor
US20070071790A1 (en) * 2005-09-28 2007-03-29 Northwestern University Biodegradable nanocomposites with enhance mechanical properties for soft tissue
WO2008112226A2 (en) * 2007-03-13 2008-09-18 Massachusetts Institute Of Technology Cre-lox based gene knockdown constructs and methods of use thereof
US20120315322A1 (en) * 2011-06-09 2012-12-13 Nnanoaxis, Llc Nanoparticle mediated gene therapy and therapeutic products for alzheimers
US20150093365A1 (en) * 2012-04-18 2015-04-02 Dana-Farber Cancer Institute, Inc. Photoactivatable caged tamoxifen and tamoxifen derivative molecules and methods of use thereof
WO2015185691A1 (en) * 2014-06-05 2015-12-10 Eth Zurich Low-leakage cellular biosensor system
US20160022835A1 (en) * 2013-03-15 2016-01-28 The Brigham And Women's Hospital, Inc. Targeted Polymeric Inflammation-Resolving Nanoparticles
US20160175462A1 (en) * 2013-06-17 2016-06-23 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002028175A2 (en) * 2000-10-03 2002-04-11 Association Pour Le Developpement De La Recherche En Genetique Moleculaire (Aderegem) Transgenic mouse for targeted recombination mediated by modified cre-er
US20040023203A1 (en) * 2002-05-31 2004-02-05 Gero Miesenbock Heterologous stimulus-gated ion channels and methods of using same
US20050013869A1 (en) * 2003-07-18 2005-01-20 Chaw Cheng Shu Sustained release formulation for carbamates and a method therefor
US20070071790A1 (en) * 2005-09-28 2007-03-29 Northwestern University Biodegradable nanocomposites with enhance mechanical properties for soft tissue
WO2008112226A2 (en) * 2007-03-13 2008-09-18 Massachusetts Institute Of Technology Cre-lox based gene knockdown constructs and methods of use thereof
US20120315322A1 (en) * 2011-06-09 2012-12-13 Nnanoaxis, Llc Nanoparticle mediated gene therapy and therapeutic products for alzheimers
US20150093365A1 (en) * 2012-04-18 2015-04-02 Dana-Farber Cancer Institute, Inc. Photoactivatable caged tamoxifen and tamoxifen derivative molecules and methods of use thereof
US20160022835A1 (en) * 2013-03-15 2016-01-28 The Brigham And Women's Hospital, Inc. Targeted Polymeric Inflammation-Resolving Nanoparticles
US20160175462A1 (en) * 2013-06-17 2016-06-23 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
WO2015185691A1 (en) * 2014-06-05 2015-12-10 Eth Zurich Low-leakage cellular biosensor system

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BLUM ET AL.: "Prevention of K-Ras- and Pten-mediated intravaginal tumors by treatment with camptothecin-loaded PLGA nanoparticles", DRUG DELIVERY AND TRANSLATIONAL RESEARCH, vol. 1, no. 5, 10 September 2011 (2011-09-10), pages 383 - 394, XP055534589 *
ICHISE ET AL.: "Establishment of a tamoxifen-inducible Cre-driver mouse strain for widespread and temporal genetic modification in adult mice", EXPERIMENTAL ANIMALS, vol. 65, no. 3, 29 July 2016 (2016-07-29), pages 231 - 244, XP055534554 *
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