BR112015031194A2 - Molécula quimérica e método para fazer a mesma, polinucleotídeo ou um conjunto de polinucleotídeos, vetor ou um conjunto de vetores, célula hospedeira, composição farmacêutica, uso dos anteriores, e método para aumentar a atividade de fviii da molécula quimérica - Google Patents

Molécula quimérica e método para fazer a mesma, polinucleotídeo ou um conjunto de polinucleotídeos, vetor ou um conjunto de vetores, célula hospedeira, composição farmacêutica, uso dos anteriores, e método para aumentar a atividade de fviii da molécula quimérica

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Publication number
BR112015031194A2
BR112015031194A2 BR112015031194A BR112015031194A BR112015031194A2 BR 112015031194 A2 BR112015031194 A2 BR 112015031194A2 BR 112015031194 A BR112015031194 A BR 112015031194A BR 112015031194 A BR112015031194 A BR 112015031194A BR 112015031194 A2 BR112015031194 A2 BR 112015031194A2
Authority
BR
Brazil
Prior art keywords
chimeric molecule
vectors
polynucleotides
polynucleotide
vector
Prior art date
Application number
BR112015031194A
Other languages
English (en)
Other versions
BR112015031194B1 (pt
Inventor
Seth Chhabra Ekta
Kulman John
Liu Tongyao
Original Assignee
Biogen Ma Inc
Bioverativ Therapeutics Inc
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 Biogen Ma Inc, Bioverativ Therapeutics Inc filed Critical Biogen Ma Inc
Publication of BR112015031194A2 publication Critical patent/BR112015031194A2/pt
Publication of BR112015031194B1 publication Critical patent/BR112015031194B1/pt

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    • 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
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • 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
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/35Fusion polypeptide containing a fusion for enhanced stability/folding during expression, e.g. fusions with chaperones or thioredoxin
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/90Fusion polypeptide containing a motif for post-translational modification

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Biotechnology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

MOLÉCULA QUIMÉRICA E MÉTODO PARA FAZER A MESMA, POLINUCLEOTÍDEO OU UM CONJUNTO DE POLINUCLEOTÍDEOS, VETOR OU UM CONJUNTO DE VETORES, CÉLULA HOSPEDEIRA, COMPOSIÇÃO FARMACÊUTICA, USO DOS ANTERIORES, E MÉTODO PARA AUMENTAR A ATIVIDADE DE FVIII DA MOLÉCULA QUIMÉRICA. A presente invenção refere-se a uma molécula quimérica, composta por uma proteína VWF fundida a uma porção heteróloga através de um ligante VWF. A invenção fornece um ligante VWF eficiente que pode ser clivado na presença de trombina. A molécula quimérica pode também compreender uma cadeia polipeptídica composta por uma proteína FVIII e uma segunda porção heteróloga, em que a cadeia compreendendo a proteína VWF e a proteína FVIII estão associadas uma à outra. A invenção também inclui nucleotídeos, vetores, células hospedeiras e métodos para uso das proteínas quiméricas.
BR112015031194-6A 2013-06-28 2014-06-27 Molécula quimérica, composição farmacêutica, e uso dos anteriores BR112015031194B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361840872P 2013-06-28 2013-06-28
US61/840,872 2013-06-28
PCT/US2014/044731 WO2014210558A1 (en) 2013-06-28 2014-06-27 Thrombin cleavable linker with xten and its uses thereof

Publications (2)

Publication Number Publication Date
BR112015031194A2 true BR112015031194A2 (pt) 2017-09-19
BR112015031194B1 BR112015031194B1 (pt) 2023-07-18

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Also Published As

Publication number Publication date
SG10201913738YA (en) 2020-03-30
SG11201509313PA (en) 2016-01-28
CN105392495A (zh) 2016-03-09
US20160251408A1 (en) 2016-09-01
JP2022000053A (ja) 2022-01-04
KR20160023654A (ko) 2016-03-03
IL266462B2 (en) 2023-04-01
PH12015502614A1 (en) 2016-02-29
TWI716340B (zh) 2021-01-21
TW202045535A (zh) 2020-12-16
AU2014302100A1 (en) 2015-11-26
HK1223302A1 (zh) 2017-07-28
AU2020203063B2 (en) 2023-05-25
TW201514204A (zh) 2015-04-16
EA201592022A1 (ru) 2016-05-31
JP2021072853A (ja) 2021-05-13
CN113817069A (zh) 2021-12-21
CL2015003710A1 (es) 2016-08-26
KR20240023705A (ko) 2024-02-22
TWI770467B (zh) 2022-07-11
SG10201710616XA (en) 2018-02-27
WO2014210558A1 (en) 2014-12-31
KR20230007555A (ko) 2023-01-12
EP4368194A2 (en) 2024-05-15
CN113831415A (zh) 2021-12-24
IL242436B (en) 2019-05-30
IL297611A (en) 2022-12-01
TW202313672A (zh) 2023-04-01
EP3013358A4 (en) 2017-03-22
US20220106383A1 (en) 2022-04-07
JP7297837B2 (ja) 2023-06-26
NZ713904A (en) 2022-02-25
JP2023107932A (ja) 2023-08-03
AU2023219840A1 (en) 2023-09-14
AU2014302100B2 (en) 2020-02-13
AU2020203063A1 (en) 2020-05-28
JP7005800B2 (ja) 2022-02-10
EP3013358A1 (en) 2016-05-04
KR102666819B1 (ko) 2024-05-23
MX2015016567A (es) 2016-03-31
IL266462B (en) 2022-12-01
JP2019010124A (ja) 2019-01-24
IL266462A (en) 2019-06-30
US20240083975A1 (en) 2024-03-14
JP2016523919A (ja) 2016-08-12
CA2913078A1 (en) 2014-12-31

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B25A Requested transfer of rights approved

Owner name: BIOVERATIV THERAPEUTICS INC. (US)

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

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