CL2020001611A1 - Polipéptidos y vectores virales que codifican variantes de fviii recombinantes con mayor expresión para la terapia génica de hemofilia a. (divisional solicitud 201801301) - Google Patents

Polipéptidos y vectores virales que codifican variantes de fviii recombinantes con mayor expresión para la terapia génica de hemofilia a. (divisional solicitud 201801301)

Info

Publication number
CL2020001611A1
CL2020001611A1 CL2020001611A CL2020001611A CL2020001611A1 CL 2020001611 A1 CL2020001611 A1 CL 2020001611A1 CL 2020001611 A CL2020001611 A CL 2020001611A CL 2020001611 A CL2020001611 A CL 2020001611A CL 2020001611 A1 CL2020001611 A1 CL 2020001611A1
Authority
CL
Chile
Prior art keywords
gene therapy
polypeptides
viral vectors
vectors encoding
expressing recombinant
Prior art date
Application number
CL2020001611A
Other languages
English (en)
Inventor
Falko-Günter Falkner
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Baxalta Inc
Baxalta GmbH
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 Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of CL2020001611A1 publication Critical patent/CL2020001611A1/es

Links

Classifications

    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente descripción proporciona, entre otros aspectos, polinucleótidos con alteración por codones que codifican variantes de Factor VIII para su expresión en células de mamífero. En algunas modalidades, la descripción proporciona también vectores de terapia génica de mamífero y métodos para el tratamiento de la hemofilia A.
CL2020001611A 2015-11-13 2020-06-16 Polipéptidos y vectores virales que codifican variantes de fviii recombinantes con mayor expresión para la terapia génica de hemofilia a. (divisional solicitud 201801301) CL2020001611A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13

Publications (1)

Publication Number Publication Date
CL2020001611A1 true CL2020001611A1 (es) 2020-10-30

Family

ID=57539606

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2018001301A CL2018001301A1 (es) 2015-11-13 2018-05-14 Vectores virales que codifican variantes fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a.
CL2020001611A CL2020001611A1 (es) 2015-11-13 2020-06-16 Polipéptidos y vectores virales que codifican variantes de fviii recombinantes con mayor expresión para la terapia génica de hemofilia a. (divisional solicitud 201801301)

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CL2018001301A CL2018001301A1 (es) 2015-11-13 2018-05-14 Vectores virales que codifican variantes fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a.

Country Status (17)

Country Link
US (1) US10189889B2 (es)
EP (1) EP3374388A1 (es)
JP (1) JP6695426B2 (es)
KR (1) KR102404550B1 (es)
CN (1) CN108602876B (es)
AU (1) AU2016354550B2 (es)
BR (1) BR112018009732A8 (es)
CA (1) CA3005565A1 (es)
CL (2) CL2018001301A1 (es)
CO (1) CO2018005380A2 (es)
EA (2) EA202092049A1 (es)
IL (1) IL259295B1 (es)
MX (1) MX2018005969A (es)
SG (1) SG11201804064WA (es)
TW (2) TWI823830B (es)
WO (1) WO2017083764A1 (es)
ZA (1) ZA201803442B (es)

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MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
CN109735547B (zh) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 一种提高毕赤酵母外源蛋白表达量的启动子及其应用
US20200405883A1 (en) * 2019-06-20 2020-12-31 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用

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Also Published As

Publication number Publication date
SG11201804064WA (en) 2018-06-28
BR112018009732A8 (pt) 2019-02-26
US20170226188A1 (en) 2017-08-10
EA202092049A1 (ru) 2020-11-10
JP6695426B2 (ja) 2020-05-20
KR102404550B1 (ko) 2022-05-31
CO2018005380A2 (es) 2018-05-31
AU2016354550A1 (en) 2018-06-07
US10189889B2 (en) 2019-01-29
CN108602876B (zh) 2022-07-05
NZ742555A (en) 2023-12-22
TW202229555A (zh) 2022-08-01
ZA201803442B (en) 2019-07-31
IL259295B1 (en) 2024-06-01
CA3005565A1 (en) 2017-05-18
AU2016354550B2 (en) 2020-04-16
MX2018005969A (es) 2018-11-29
EP3374388A1 (en) 2018-09-19
KR20180070700A (ko) 2018-06-26
EA201891138A1 (ru) 2018-12-28
CL2018001301A1 (es) 2018-12-28
CN108602876A (zh) 2018-09-28
EA036944B1 (ru) 2021-01-19
TWI823830B (zh) 2023-12-01
TW201723180A (zh) 2017-07-01
WO2017083764A1 (en) 2017-05-18
IL259295A (en) 2018-07-31
JP2018537089A (ja) 2018-12-20
BR112018009732A2 (pt) 2018-11-21

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