MX2021008550A - Vectores viricos que codifican variantes del factor anti-hemofilico a (fviii) recombinantes con mayor expresion para la genoterapia de la hemofilia a. - Google Patents
Vectores viricos que codifican variantes del factor anti-hemofilico a (fviii) recombinantes con mayor expresion para la genoterapia de la hemofilia a.Info
- Publication number
- MX2021008550A MX2021008550A MX2021008550A MX2021008550A MX2021008550A MX 2021008550 A MX2021008550 A MX 2021008550A MX 2021008550 A MX2021008550 A MX 2021008550A MX 2021008550 A MX2021008550 A MX 2021008550A MX 2021008550 A MX2021008550 A MX 2021008550A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- hemophilia
- viral vectors
- increased expression
- vectors encoding
- Prior art date
Links
Classifications
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- 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
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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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
-
- 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
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- 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
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- 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)
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- 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
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
La presente descripción proporciona, entre otros aspectos, polinucleótidos modificados por codones que codifican variantes del Factor VIII para su expresión en células de mamíferos. En algunas modalidades, la descripción proporciona también vectores para genoterapia en mamíferos y métodos para el tratamiento de la hemofilia A.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962793058P | 2019-01-16 | 2019-01-16 | |
PCT/US2020/013722 WO2020150375A1 (en) | 2019-01-16 | 2020-01-15 | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021008550A true MX2021008550A (es) | 2021-08-19 |
Family
ID=69593774
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021008550A MX2021008550A (es) | 2019-01-16 | 2020-01-15 | Vectores viricos que codifican variantes del factor anti-hemofilico a (fviii) recombinantes con mayor expresion para la genoterapia de la hemofilia a. |
Country Status (19)
Country | Link |
---|---|
US (2) | US11707536B2 (es) |
EP (1) | EP3911672A1 (es) |
JP (1) | JP2022517267A (es) |
KR (1) | KR20210117291A (es) |
CN (1) | CN113614104A (es) |
AR (1) | AR117825A1 (es) |
AU (1) | AU2020208375A1 (es) |
BR (1) | BR112021013874A2 (es) |
CA (1) | CA3127065A1 (es) |
CL (1) | CL2021001880A1 (es) |
CO (1) | CO2021009338A2 (es) |
EA (1) | EA202191977A1 (es) |
EC (1) | ECSP21060605A (es) |
IL (1) | IL284648A (es) |
MX (1) | MX2021008550A (es) |
PE (1) | PE20220429A1 (es) |
SG (1) | SG11202107421YA (es) |
TW (1) | TW202039546A (es) |
WO (1) | WO2020150375A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2022211791A1 (en) * | 2021-03-30 | 2022-10-06 | AAVnerGene Inc. | Modified plasma clotting factor viii and method of use thereof |
US11795207B2 (en) | 2021-03-30 | 2023-10-24 | AAVnerGene Inc. | Modified plasma clotting factor VIII and method of use thereof |
CN116710554A (zh) * | 2021-11-25 | 2023-09-05 | 四川至善唯新生物科技有限公司 | 基因工程改造获得的分泌能力和凝血活性增强的人类八因子 |
US20230374546A1 (en) * | 2022-03-11 | 2023-11-23 | Homology Medicines, Inc. | Bidirectional dual promoter expression vectors and uses thereof |
Family Cites Families (50)
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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 |
US5543502A (en) | 1986-06-24 | 1996-08-06 | 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 |
JPH08511423A (ja) | 1993-06-10 | 1996-12-03 | ジェネティック セラピー,インコーポレイテッド | 血友病治療のためのアデノウイルスベクター |
SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
US6818439B1 (en) | 1994-12-30 | 2004-11-16 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders |
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 |
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US20060099685A1 (en) | 1999-04-15 | 2006-05-11 | Yallop Christopher A | Recombinant expression of factor VIII in human cells |
EP1572889B1 (en) | 2001-10-05 | 2008-12-17 | Expression Therapeutics, LLC | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
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CA2864879C (en) | 2012-02-17 | 2021-07-20 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
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US10398787B2 (en) | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
AU2013336601B2 (en) | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
SI3889173T1 (sl) | 2013-02-15 | 2023-11-30 | Bioverativ Therapeutics Inc. | Optimiran gen dejavnika VIII |
CA2905739A1 (en) | 2013-03-15 | 2014-09-25 | Bayer Healthcare Llc | Recombinant factor viii formulations |
PL3044231T3 (pl) | 2013-09-12 | 2021-01-11 | Biomarin Pharmaceutical Inc. | Wektory aav zawierające gen kodujący czynnik viii |
EP3180022A4 (en) | 2014-08-13 | 2019-02-20 | The Children's Hospital of Philadelphia | IMPROVED EXPRESSION CASSETTE FOR THE PACKAGING AND EXPRESSION OF VARIANT FACTOR VIII FOR THE TREATMENT OF HEMOSTATIC DISEASES |
US10308705B2 (en) | 2015-02-06 | 2019-06-04 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor VIII gene expression cassettes and their use |
EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
SG10202106307UA (en) | 2015-11-13 | 2021-07-29 | Takeda Pharmaceuticals Co | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
US20210403948A1 (en) * | 2018-10-26 | 2021-12-30 | Vrije Universiteit Brussel | Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof |
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2020
- 2020-01-13 TW TW109101061A patent/TW202039546A/zh unknown
- 2020-01-15 EP EP20705834.8A patent/EP3911672A1/en active Pending
- 2020-01-15 PE PE2021001192A patent/PE20220429A1/es unknown
- 2020-01-15 SG SG11202107421YA patent/SG11202107421YA/en unknown
- 2020-01-15 EA EA202191977A patent/EA202191977A1/ru unknown
- 2020-01-15 CA CA3127065A patent/CA3127065A1/en active Pending
- 2020-01-15 AU AU2020208375A patent/AU2020208375A1/en active Pending
- 2020-01-15 MX MX2021008550A patent/MX2021008550A/es unknown
- 2020-01-15 BR BR112021013874-9A patent/BR112021013874A2/pt unknown
- 2020-01-15 US US16/743,850 patent/US11707536B2/en active Active
- 2020-01-15 JP JP2021541022A patent/JP2022517267A/ja active Pending
- 2020-01-15 KR KR1020217025460A patent/KR20210117291A/ko unknown
- 2020-01-15 WO PCT/US2020/013722 patent/WO2020150375A1/en active Application Filing
- 2020-01-15 CN CN202080014260.8A patent/CN113614104A/zh active Pending
- 2020-01-16 AR ARP200100113A patent/AR117825A1/es unknown
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2021
- 2021-07-06 IL IL284648A patent/IL284648A/en unknown
- 2021-07-15 CL CL2021001880A patent/CL2021001880A1/es unknown
- 2021-07-16 CO CONC2021/0009338A patent/CO2021009338A2/es unknown
- 2021-08-16 EC ECSENADI202160605A patent/ECSP21060605A/es unknown
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2022
- 2022-12-22 US US18/145,784 patent/US20230211017A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CN113614104A (zh) | 2021-11-05 |
PE20220429A1 (es) | 2022-03-29 |
US20200289672A1 (en) | 2020-09-17 |
EA202191977A1 (ru) | 2021-12-15 |
CL2021001880A1 (es) | 2022-01-14 |
KR20210117291A (ko) | 2021-09-28 |
BR112021013874A2 (pt) | 2021-09-21 |
SG11202107421YA (en) | 2021-08-30 |
TW202039546A (zh) | 2020-11-01 |
US11707536B2 (en) | 2023-07-25 |
CA3127065A1 (en) | 2020-07-23 |
WO2020150375A1 (en) | 2020-07-23 |
EP3911672A1 (en) | 2021-11-24 |
US20230211017A1 (en) | 2023-07-06 |
IL284648A (en) | 2021-08-31 |
JP2022517267A (ja) | 2022-03-07 |
CO2021009338A2 (es) | 2021-07-30 |
AR117825A1 (es) | 2021-08-25 |
AU2020208375A1 (en) | 2021-07-29 |
ECSP21060605A (es) | 2022-01-31 |
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