CO2023006605A2 - Variante de adamts13 que tiene actividad o velocidad de escape incrementada contra un autoanticuerpo - Google Patents

Variante de adamts13 que tiene actividad o velocidad de escape incrementada contra un autoanticuerpo

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Publication number
CO2023006605A2
CO2023006605A2 CONC2023/0006605A CO2023006605A CO2023006605A2 CO 2023006605 A2 CO2023006605 A2 CO 2023006605A2 CO 2023006605 A CO2023006605 A CO 2023006605A CO 2023006605 A2 CO2023006605 A2 CO 2023006605A2
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CO
Colombia
Prior art keywords
adamts13
autoantibody
present
autoantibodies
novel
Prior art date
Application number
CONC2023/0006605A
Other languages
English (en)
Inventor
Hyun-Ja Nam
Sung Ho Hwang
Heechun Kwak
Gahee Choi
Suyong Kim
Yu Young Kim
Yongmin Lee
Chae Mok Lee
Sunhye Shin
Young Eun Kwon
Seunghyun Jo
Original Assignee
Green Cross Corp
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.)
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Publication date
Application filed by Green Cross Corp filed Critical Green Cross Corp
Publication of CO2023006605A2 publication Critical patent/CO2023006605A2/es

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6489Metalloendopeptidases (3.4.24)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • 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
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4886Metalloendopeptidases (3.4.24), e.g. collagenase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • CCHEMISTRY; METALLURGY
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    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/24Metalloendopeptidases (3.4.24)
    • C12Y304/24087ADAMTS13 endopeptidase (3.4.24.87)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6854Immunoglobulins
    • 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
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/30Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/34Identification of a linear epitope shorter than 20 amino acid residues or of a conformational epitope defined by amino acid residues
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/948Hydrolases (3) acting on peptide bonds (3.4)
    • G01N2333/95Proteinases, i.e. endopeptidases (3.4.21-3.4.99)
    • G01N2333/964Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue
    • G01N2333/96402Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from non-mammals
    • G01N2333/96405Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from non-mammals in general
    • G01N2333/96408Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from non-mammals in general with EC number
    • G01N2333/96419Metalloendopeptidases (3.4.24)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
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    • G01N2333/95Proteinases, i.e. endopeptidases (3.4.21-3.4.99)
    • G01N2333/964Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue
    • G01N2333/96425Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals
    • G01N2333/96427Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general
    • G01N2333/9643Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general with EC number
    • G01N2333/96486Metalloendopeptidases (3.4.24)
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    • G01N33/564Immunoassay; Biospecific binding assay; Materials therefor for pre-existing immune complex or autoimmune disease, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9

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Abstract

La presente invención se refiere a una proteína mutante ADAMTS13 que tiene una velocidad de escape mejorada frente a un autoanticuerpo y a una composición para prevenir o tratar enfermedades trombóticas utilizando la misma. Al evitar de manera eficiente los autoanticuerpos representativos que se sabe que tienen una alta afinidad de unión al dominio principal de ADAMTS13, la proteína variante de ADAMTS13 de la presente invención se puede usar como una composición terapéutica eficaz para varias enfermedades trombóticas, TALES como TTP (púrpura trombocitopénica trombótica), etc., en donde la presencia de tales autoanticuerpos es la etiología principal, y puede mantener de forma estable la actividad biológica de los mismos cuando se administran en un organismo. Además, como se identifica un nuevo sitio reconocido por un autoanticuerpo dentro de ADAMTS13, la presente invención se puede usar de manera útil en la detección de nuevas variantes de ADAMTS13 que tienen una velocidad de escape de autoanticuerpos mejorada mediante la aplicación de una combinación de varias mutaciones dentro del sitio correspondiente.
CONC2023/0006605A 2020-11-18 2023-05-19 Variante de adamts13 que tiene actividad o velocidad de escape incrementada contra un autoanticuerpo CO2023006605A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
KR20200154710 2020-11-18
PCT/KR2021/014991 WO2022108148A1 (ko) 2020-11-18 2021-10-25 자가 항체에 대한 회피율 또는 활성이 증가된 adamts13 변이체

Publications (1)

Publication Number Publication Date
CO2023006605A2 true CO2023006605A2 (es) 2023-05-29

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ID=81709197

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Application Number Title Priority Date Filing Date
CONC2023/0006605A CO2023006605A2 (es) 2020-11-18 2023-05-19 Variante de adamts13 que tiene actividad o velocidad de escape incrementada contra un autoanticuerpo

Country Status (12)

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US (1) US20230405096A1 (es)
EP (1) EP4249594A4 (es)
JP (1) JP2023549878A (es)
KR (1) KR20220068144A (es)
CN (1) CN116745426A (es)
AU (1) AU2021382506A1 (es)
CA (1) CA3201986A1 (es)
CO (1) CO2023006605A2 (es)
IL (1) IL302984A (es)
MX (1) MX2023005661A (es)
WO (1) WO2022108148A1 (es)
ZA (1) ZA202305424B (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023235964A1 (en) * 2022-06-06 2023-12-14 Mcmaster University Thrombolytic protease resistant adamts13 mutants

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2382104T3 (es) * 2005-06-17 2012-06-05 Baxter International Inc. Composiciones con actividad trombolítica que comprenden ADAMTS13
JP5254973B2 (ja) 2007-06-22 2013-08-07 一般財団法人化学及血清療法研究所 新規adamts−13改変体
WO2012083370A1 (en) * 2010-12-22 2012-06-28 Cephalon Australia Pty Ltd Modified antibody with improved half-life
WO2013096793A1 (en) * 2011-12-21 2013-06-27 The Children's Hospital Of Philadelphia Gain-of-function adamts13 variants resistant to autoantibody inhibition and methods of use thereof
AU2013203062C1 (en) * 2013-03-15 2018-06-28 Takeda Pharmaceutical Company Limited Subcutaneous administration of adamts13
WO2017055908A1 (en) * 2015-09-28 2017-04-06 Noris Marina Methods of treating adamts13 deficiencies and congenital thrombotic thrombocytopenia in pediatric patients

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EP4249594A4 (en) 2024-10-23
IL302984A (en) 2023-07-01
US20230405096A1 (en) 2023-12-21
KR20220068144A (ko) 2022-05-25
CN116745426A (zh) 2023-09-12
ZA202305424B (en) 2024-09-25
EP4249594A1 (en) 2023-09-27
WO2022108148A1 (ko) 2022-05-27
JP2023549878A (ja) 2023-11-29
MX2023005661A (es) 2023-05-26
CA3201986A1 (en) 2022-05-27
AU2021382506A1 (en) 2023-06-29

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