CO2021000468A2 - Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión - Google Patents
Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresiónInfo
- Publication number
- CO2021000468A2 CO2021000468A2 CONC2021/0000468A CO2021000468A CO2021000468A2 CO 2021000468 A2 CO2021000468 A2 CO 2021000468A2 CO 2021000468 A CO2021000468 A CO 2021000468A CO 2021000468 A2 CO2021000468 A2 CO 2021000468A2
- Authority
- CO
- Colombia
- Prior art keywords
- hemophilia
- gene therapy
- viral vectors
- vectors encoding
- higher expression
- Prior art date
Links
- 208000009292 Hemophilia A Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 208000031220 Hemophilia Diseases 0.000 title 1
- 239000013603 viral vector Substances 0.000 title 1
- 108091033319 polynucleotide Proteins 0.000 abstract 3
- 102000040430 polynucleotide Human genes 0.000 abstract 3
- 239000002157 polynucleotide Substances 0.000 abstract 3
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 2
- 108010054218 Factor VIII Proteins 0.000 abstract 2
- 102000001690 Factor VIII Human genes 0.000 abstract 2
- 201000003542 Factor VIII deficiency Diseases 0.000 abstract 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 2
- 229960000301 factor viii Drugs 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 108020004705 Codon Proteins 0.000 abstract 1
- 210000004962 mammalian cell Anatomy 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- 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
-
- 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
- C12N15/861—Adenoviral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61K48/0083—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 administration regime
-
- 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
- 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)
-
- 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
-
- 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
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0381—Animal model for diseases of the hematopoietic system
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
RESUMEN La presente descripción proporciona, entre otros aspectos, polinucleótidos modificados con codones que codifican variantes del Factor VIII para su expresión en células de mamíferos. En algunas modalidades, la descripción también proporciona vectores de terapia génica de mamíferos y métodos para tratar la hemofilia A. En algunas modalidades, la presente descripción proporciona métodos para la dosificación de un paciente con hemofilia A con un polinucleótido, p. ej., un polinucleótido modificado con codones, que codifica un polipéptido del Factor VIII.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862698680P | 2018-07-16 | 2018-07-16 | |
US201962867171P | 2019-06-26 | 2019-06-26 | |
PCT/US2019/041802 WO2020018419A1 (en) | 2018-07-16 | 2019-07-15 | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021000468A2 true CO2021000468A2 (es) | 2021-04-08 |
Family
ID=67660451
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0000468A CO2021000468A2 (es) | 2018-07-16 | 2021-01-19 | Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220333135A1 (es) |
EP (1) | EP3823985A1 (es) |
JP (1) | JP2021530524A (es) |
KR (1) | KR20210034013A (es) |
CN (1) | CN112449640A (es) |
AU (1) | AU2019306194A1 (es) |
BR (1) | BR112021000695A2 (es) |
CA (1) | CA3106590A1 (es) |
CO (1) | CO2021000468A2 (es) |
MX (1) | MX2021000582A (es) |
TW (1) | TW202006141A (es) |
WO (1) | WO2020018419A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2023506171A (ja) * | 2019-12-12 | 2023-02-15 | 武田薬品工業株式会社 | 発現が上昇した組換えfviiiバリアントをコードするウイルスベクターを使用する、血友病aの遺伝子療法 |
CN117858895A (zh) * | 2021-06-14 | 2024-04-09 | 武田药品工业株式会社 | 使用表达增强的编码重组fviii变体的病毒载体的血友病a基因疗法 |
Family Cites Families (28)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US4868112A (en) | 1985-04-12 | 1989-09-19 | Genetics Institute, Inc. | Novel procoagulant proteins |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
US5610278A (en) | 1986-06-24 | 1997-03-11 | Novo Nordisk A/S | Process for producing a coagulation active complex of factor VIII fragments |
US6060447A (en) | 1987-05-19 | 2000-05-09 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
US5171844A (en) | 1987-06-12 | 1992-12-15 | Gist-Brocades N.W. | Proteins with factor viii activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
US6346513B1 (en) | 1987-06-12 | 2002-02-12 | Baxter Trading Gmbh | Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
SE9503380D0 (sv) | 1995-09-29 | 1995-09-29 | Pharmacia Ab | Protein derivatives |
US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
US7041635B2 (en) | 2003-01-28 | 2006-05-09 | In2Gen Co., Ltd. | Factor VIII polypeptide |
US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
WO2008069942A2 (en) | 2006-12-05 | 2008-06-12 | Biogen Idec Ma Inc. | Novel methods of enhancing delivery of a gene therapy vector using steroids |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
SG10201604810UA (en) * | 2012-02-07 | 2016-08-30 | Global Bio Therapeutics Inc | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
ES2959747T3 (es) | 2013-02-15 | 2024-02-28 | Bioverativ Therapeutics Inc | Gen del factor VIII optimizado |
EP2796145B1 (en) * | 2013-04-22 | 2017-11-01 | CSL Ltd. | A covalent complex of von willebrand factor and faktor viii linked by a disulphide bridge |
EP3770169A1 (en) | 2013-09-12 | 2021-01-27 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
MA42934A (fr) * | 2015-09-24 | 2018-08-01 | Biomarin Pharm Inc | Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci |
TWI777175B (zh) * | 2015-11-13 | 2022-09-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體之病毒載體 |
-
2019
- 2019-07-15 MX MX2021000582A patent/MX2021000582A/es unknown
- 2019-07-15 EP EP19755458.7A patent/EP3823985A1/en active Pending
- 2019-07-15 JP JP2021502571A patent/JP2021530524A/ja active Pending
- 2019-07-15 US US17/296,944 patent/US20220333135A1/en active Pending
- 2019-07-15 KR KR1020217003882A patent/KR20210034013A/ko unknown
- 2019-07-15 BR BR112021000695-8A patent/BR112021000695A2/pt not_active Application Discontinuation
- 2019-07-15 AU AU2019306194A patent/AU2019306194A1/en active Pending
- 2019-07-15 WO PCT/US2019/041802 patent/WO2020018419A1/en unknown
- 2019-07-15 TW TW108124947A patent/TW202006141A/zh unknown
- 2019-07-15 CA CA3106590A patent/CA3106590A1/en active Pending
- 2019-07-15 CN CN201980047074.1A patent/CN112449640A/zh active Pending
-
2021
- 2021-01-19 CO CONC2021/0000468A patent/CO2021000468A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
MX2021000582A (es) | 2021-04-12 |
WO2020018419A1 (en) | 2020-01-23 |
US20220333135A1 (en) | 2022-10-20 |
JP2021530524A (ja) | 2021-11-11 |
CN112449640A (zh) | 2021-03-05 |
AU2019306194A1 (en) | 2021-02-04 |
TW202006141A (zh) | 2020-02-01 |
CA3106590A1 (en) | 2020-01-23 |
KR20210034013A (ko) | 2021-03-29 |
BR112021000695A2 (pt) | 2021-04-20 |
EP3823985A1 (en) | 2021-05-26 |
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