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WO2002007752A3 - Red blood cell as vehicle for agent-membrane translocation sequence conjugate - Google Patents

Red blood cell as vehicle for agent-membrane translocation sequence conjugate Download PDF

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Publication number
WO2002007752A3
WO2002007752A3 PCT/GB2001/003327 GB0103327W WO0207752A3 WO 2002007752 A3 WO2002007752 A3 WO 2002007752A3 GB 0103327 W GB0103327 W GB 0103327W WO 0207752 A3 WO0207752 A3 WO 0207752A3
Authority
WO
WIPO (PCT)
Prior art keywords
red blood
blood cell
agent
vehicle
membrane translocation
Prior art date
Application number
PCT/GB2001/003327
Other languages
French (fr)
Other versions
WO2002007752A2 (en
Inventor
Roger Craig
Original Assignee
Gendel Ltd
Roger Craig
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
Priority claimed from PCT/GB2000/002848 external-priority patent/WO2001007011A1/en
Priority claimed from PCT/GB2000/003056 external-priority patent/WO2001058431A1/en
Priority claimed from US09/785,802 external-priority patent/US20020151004A1/en
Application filed by Gendel Ltd, Roger Craig filed Critical Gendel Ltd
Priority to AU2001272670A priority Critical patent/AU2001272670A1/en
Priority to MXPA03000765A priority patent/MXPA03000765A/en
Priority to CA002412544A priority patent/CA2412544A1/en
Priority to EP01951821A priority patent/EP1355656A2/en
Priority to AU2002219391A priority patent/AU2002219391A1/en
Priority to PCT/GB2002/000169 priority patent/WO2002057436A2/en
Publication of WO2002007752A2 publication Critical patent/WO2002007752A2/en
Publication of WO2002007752A3 publication Critical patent/WO2002007752A3/en

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    • 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
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/18Erythrocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005Wall or coating material
    • A61K9/5063Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068Cell membranes or bacterial membranes enclosing drugs
    • 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
    • C07K14/43504Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • C07K14/43595Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates from coelenteratae, e.g. medusae
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • 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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/721Steroid/thyroid hormone superfamily, e.g. GR, EcR, androgen receptor, oestrogen receptor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0641Erythrocytes
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/01Animal expressing industrially exogenous proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
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    • C12N2517/00Cells related to new breeds of animals
    • C12N2517/02Cells from transgenic animals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Botany (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

We describe a method of preparing a red blood cell vehicle suitable for delivering an agent to a target site in a vertebrate, the method comprising the steps of: (a) providing a red blood cell; and (b) loading the red blood cell with an agent-MTS conjugate.
PCT/GB2001/003327 2000-07-24 2001-07-24 Red blood cell as vehicle for agent-membrane translocation sequence conjugate WO2002007752A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
AU2001272670A AU2001272670A1 (en) 2000-07-24 2001-07-24 Polypeptide delivery II-2
MXPA03000765A MXPA03000765A (en) 2000-07-24 2001-07-24 Polypeptide delivery ii-2.
CA002412544A CA2412544A1 (en) 2000-07-24 2001-07-24 Polypeptide delivery ii-2
EP01951821A EP1355656A2 (en) 2000-07-24 2001-07-24 Red blood cell as vehicle for agent-membrane translocation sequence conjugate
AU2002219391A AU2002219391A1 (en) 2001-01-19 2002-01-16 Red blood cell from a transgenic animal as vehicle for polypeptide delivery
PCT/GB2002/000169 WO2002057436A2 (en) 2001-01-19 2002-01-16 Red blood cell from a transgenic animal as vehicle for polypeptide delivery

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
PCT/GB2000/002848 WO2001007011A1 (en) 1999-07-23 2000-07-24 Method for releasing an agent from a red blood cell
GBPCT/GB00/02848 2000-07-24
GBPCT/GB00/03056 2000-08-09
PCT/GB2000/003056 WO2001058431A1 (en) 2000-02-08 2000-08-09 Method for loading a red blood cell with an agent
GB0100417 2001-02-01
GBPCT/GB01/00417 2001-02-01
US09/785,802 2001-02-16
US09/785,802 US20020151004A1 (en) 2000-07-24 2001-02-16 Delivery vehicles and methods for using the same

Publications (2)

Publication Number Publication Date
WO2002007752A2 WO2002007752A2 (en) 2002-01-31
WO2002007752A3 true WO2002007752A3 (en) 2003-08-28

Family

ID=56290173

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2001/003327 WO2002007752A2 (en) 2000-07-24 2001-07-24 Red blood cell as vehicle for agent-membrane translocation sequence conjugate

Country Status (5)

Country Link
EP (1) EP1355656A2 (en)
AU (1) AU2001272670A1 (en)
CA (1) CA2412544A1 (en)
MX (1) MXPA03000765A (en)
WO (1) WO2002007752A2 (en)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2872625B1 (en) 2012-07-11 2016-11-16 Sangamo BioSciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
EP2868326A1 (en) * 2013-11-04 2015-05-06 Université Pierre et Marie Curie (Paris 6) Peptide inhibitors of TEAD/YAP-TAZ interaction
SG11201802754XA (en) 2015-10-07 2018-05-30 Sangui Bio Pty Ltd Blood preparation and profiling
WO2017070196A1 (en) * 2015-10-21 2017-04-27 The Methodist Hospital System Cellular vectors for delivery of therapeutics
SG11201805052YA (en) * 2015-12-22 2018-07-30 Sangui Bio Pty Ltd Therapeutic methods using erythrocytes
KR102712926B1 (en) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 Methods and compositions for the treatment of Fabry disease
AU2017379367B2 (en) 2016-12-20 2023-12-07 Sangui Bio Pty. Ltd Blood profiling with protease inhibitors
WO2019112974A1 (en) * 2017-12-04 2019-06-13 The General Hospital Corporation Extracellular redox enzyme system to alleviate disease
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
EP4051322A1 (en) 2019-11-01 2022-09-07 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992021378A1 (en) * 1991-06-03 1992-12-10 Consiglio Nazionale Delle Ricerche Vaccines with erythrocytes as antigen carriers
US6015787A (en) * 1997-11-04 2000-01-18 New England Medical Center Hospitals, Inc. Cell-permeable protein inhibitors of calpain
WO2000007630A1 (en) * 1998-07-31 2000-02-17 Gendel Limited Nucleic acid delivery
WO2001007011A1 (en) * 1999-07-23 2001-02-01 Gendel Limited Method for releasing an agent from a red blood cell

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992021378A1 (en) * 1991-06-03 1992-12-10 Consiglio Nazionale Delle Ricerche Vaccines with erythrocytes as antigen carriers
US6015787A (en) * 1997-11-04 2000-01-18 New England Medical Center Hospitals, Inc. Cell-permeable protein inhibitors of calpain
WO2000007630A1 (en) * 1998-07-31 2000-02-17 Gendel Limited Nucleic acid delivery
WO2001007011A1 (en) * 1999-07-23 2001-02-01 Gendel Limited Method for releasing an agent from a red blood cell

Also Published As

Publication number Publication date
CA2412544A1 (en) 2002-01-31
WO2002007752A2 (en) 2002-01-31
AU2001272670A1 (en) 2002-02-05
MXPA03000765A (en) 2004-05-21
EP1355656A2 (en) 2003-10-29

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