CO2024001464A2 - Genes optimizados del factor viii - Google Patents
Genes optimizados del factor viiiInfo
- Publication number
- CO2024001464A2 CO2024001464A2 CONC2024/0001464A CO2024001464A CO2024001464A2 CO 2024001464 A2 CO2024001464 A2 CO 2024001464A2 CO 2024001464 A CO2024001464 A CO 2024001464A CO 2024001464 A2 CO2024001464 A2 CO 2024001464A2
- Authority
- CO
- Colombia
- Prior art keywords
- factor viii
- optimized factor
- codon
- optimized
- viii genes
- Prior art date
Links
- 108010054218 Factor VIII Proteins 0.000 title abstract 4
- 102000001690 Factor VIII Human genes 0.000 abstract 3
- 229960000301 factor viii Drugs 0.000 abstract 3
- 229920001184 polypeptide Polymers 0.000 abstract 2
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 2
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 1
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
- 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
- A61K48/0058—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
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- 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
- 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
- C12N2750/14143—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
- 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
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14341—Use of virus, viral particle or viral elements as a vector
- C12N2750/14343—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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
La presente divulgación proporciona secuencias de Factor VIII con optimización de codones, vectores y células huésped que comprenden secuencias de Factor VIII con optimización de codones, polipéptidos codificados por secuencias de Factor VIII con optimización de codones y métodos para producir dichos polipéptidos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163236225P | 2021-08-23 | 2021-08-23 | |
PCT/US2022/075282 WO2023028456A1 (en) | 2021-08-23 | 2022-08-22 | Optimized factor viii genes |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2024001464A2 true CO2024001464A2 (es) | 2024-03-07 |
Family
ID=83280138
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2024/0001464A CO2024001464A2 (es) | 2021-08-23 | 2024-02-12 | Genes optimizados del factor viii |
Country Status (10)
Country | Link |
---|---|
US (1) | US20230084036A1 (es) |
KR (1) | KR20240049332A (es) |
CN (1) | CN117836319A (es) |
AR (1) | AR126846A1 (es) |
AU (1) | AU2022332276A1 (es) |
CA (1) | CA3229323A1 (es) |
CO (1) | CO2024001464A2 (es) |
IL (1) | IL310997A (es) |
TW (1) | TW202323274A (es) |
WO (1) | WO2023028456A1 (es) |
Family Cites Families (48)
Publication number | Priority date | Publication date | Assignee | Title |
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US4704362A (en) | 1977-11-08 | 1987-11-03 | Genentech, Inc. | Recombinant cloning vehicle microbial polypeptide expression |
JPH0788399B2 (ja) | 1985-04-12 | 1995-09-27 | ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド | 新規プロコアギュラント蛋白質 |
KR910006424B1 (ko) | 1985-08-21 | 1991-08-24 | 인코텍스 비.브이 | 편성브리프(brief) 제조방법 |
ATE87663T1 (de) | 1986-01-03 | 1993-04-15 | Genetics Inst | Verfahren zur herstellung von faktor-viii:c-typ- proteinen. |
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 |
EP0279582A3 (en) | 1987-02-17 | 1989-10-18 | Pharming B.V. | Dna sequences to target proteins to the mammary gland for efficient secretion |
US6060447A (en) | 1987-05-19 | 2000-05-09 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
IE69026B1 (en) | 1987-06-12 | 1996-08-07 | Immuno Ag | Novel 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 |
DE3720246A1 (de) | 1987-06-19 | 1988-12-29 | Behringwerke Ag | Faktor viii:c-aehnliches molekuel mit koagulationsaktivitaet |
FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
US5633076A (en) | 1989-12-01 | 1997-05-27 | Pharming Bv | Method of producing a transgenic bovine or transgenic bovine embryo |
SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
MX9204374A (es) | 1991-07-25 | 1993-03-01 | Idec Pharma Corp | Anticuerpo recombinante y metodo para su produccion. |
US5736137A (en) | 1992-11-13 | 1998-04-07 | Idec Pharmaceuticals Corporation | Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma |
ATE236987T1 (de) | 1992-11-13 | 2003-04-15 | Idec Pharma Corp | Konsensus-kozak-sequenzen zur säugetier- exprimierung |
SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
US5827690A (en) | 1993-12-20 | 1998-10-27 | Genzyme Transgenics Corporatiion | Transgenic production of antibodies in milk |
SE9503380D0 (sv) | 1995-09-29 | 1995-09-29 | Pharmacia Ab | Protein derivatives |
GB9524973D0 (en) | 1995-12-06 | 1996-02-07 | Lynxvale Ltd | Viral vectors |
US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
CA2225189C (en) | 1997-03-06 | 2010-05-25 | Queen's University At Kingston | Canine factor viii gene, protein and methods of use |
PT981637E (pt) | 1997-03-14 | 2005-09-30 | Biogen Idec Inc | Metodo para integrar genes em sitios especificos em celulas de mamifero atraves de recombinacao homologa e vectores para a realizacao do mesmo |
US7041635B2 (en) | 2003-01-28 | 2006-05-09 | In2Gen Co., Ltd. | Factor VIII polypeptide |
EP1616012B1 (en) | 2003-04-24 | 2007-12-19 | Fondazione Centro San Raffaele Del Monte Tabor | Synthetic bi-directional promoters and uses thereof |
EP2002003B1 (en) | 2005-05-27 | 2015-12-30 | Ospedale San Raffaele S.r.l. | Gene vector comprising mi-rna |
WO2010055413A1 (en) | 2008-11-12 | 2010-05-20 | Fondazione Centro San Raffaele Del Monte Tabor | Gene vector for inducing transgene-specific immune tolerance |
MX362028B (es) | 2009-02-03 | 2019-01-04 | Amunix Pharmaceuticals Inc | Polipeptidos recombinantes extendidos y composiciones que comprenden los mismos. |
US8703717B2 (en) | 2009-02-03 | 2014-04-22 | Amunix Operating Inc. | Growth hormone polypeptides and methods of making and using same |
US8680050B2 (en) | 2009-02-03 | 2014-03-25 | Amunix Operating Inc. | Growth hormone polypeptides fused to extended recombinant polypeptides and methods of making and using same |
PL2424571T3 (pl) | 2009-04-30 | 2020-10-19 | Ospedale San Raffaele S.R.L | Wektor genowy |
ES2705249T3 (es) | 2009-06-08 | 2019-03-22 | Amunix Operating Inc | Polipéptidos reguladores de glucosa y métodos para su producción y uso |
SG10201704777RA (en) | 2009-06-08 | 2017-07-28 | Amunix Operating Inc | Growth hormone polypeptides and methods of making and using same |
BR112012004094A2 (pt) | 2009-08-24 | 2016-03-08 | Amunix Operating Inc | composições de fator vii de coagulação e métodos para fazer e usar as mesmas |
WO2011028344A2 (en) | 2009-08-25 | 2011-03-10 | Amunix Operating Inc. | Interleukin-1 receptor antagonist compositions and methods of making and using same |
RS63870B1 (sr) | 2012-02-15 | 2023-01-31 | Bioverativ Therapeutics Inc | Sastavi faktora viii i postupci za pravljenje i upotrebu istih |
JP6383666B2 (ja) | 2012-02-15 | 2018-08-29 | バイオベラティブ セラピューティクス インコーポレイテッド | 組換え第viii因子タンパク質 |
HRP20231183T1 (hr) * | 2013-02-15 | 2024-01-05 | Bioverativ Therapeutics Inc. | Optimizirani gen faktora viii |
RU2710307C2 (ru) * | 2013-09-12 | 2019-12-26 | Байомарин Фармасьютикал Инк. | Векторы экспрессии фактора viii на основе аденоассоциированного вируса |
AU2015204646B2 (en) | 2014-01-10 | 2020-08-27 | Bioverativ Therapeutics Inc. | Factor VIII chimeric proteins and uses thereof |
TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
SI3411478T1 (sl) * | 2016-02-01 | 2022-10-28 | Bioverativ Therapeutics Inc. | Optimirani geni dejavnika VIII |
EP3476860A4 (en) * | 2016-06-24 | 2020-01-22 | Mogam Institute for Biomedical Research | RECOMBINANT SINGLE CHAIN FVIII AND CHEMICAL CONJUGATE THEREOF |
SG11202005281XA (en) * | 2017-12-06 | 2020-07-29 | Generation Bio Co | Gene editing using a modified closed-ended dna (cedna) |
UY38389A (es) * | 2018-09-27 | 2020-04-30 | Sigilon Therapeutics Inc | Dispositivos implantables para terapia celular y métodos relacionados |
US20220243201A1 (en) * | 2020-08-23 | 2022-08-04 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
US20220090130A1 (en) * | 2020-08-23 | 2022-03-24 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
-
2022
- 2022-08-22 IL IL310997A patent/IL310997A/en unknown
- 2022-08-22 TW TW111131533A patent/TW202323274A/zh unknown
- 2022-08-22 AR ARP220102260A patent/AR126846A1/es unknown
- 2022-08-22 US US17/821,359 patent/US20230084036A1/en active Pending
- 2022-08-22 CN CN202280057096.8A patent/CN117836319A/zh active Pending
- 2022-08-22 AU AU2022332276A patent/AU2022332276A1/en active Pending
- 2022-08-22 WO PCT/US2022/075282 patent/WO2023028456A1/en active Application Filing
- 2022-08-22 CA CA3229323A patent/CA3229323A1/en active Pending
- 2022-08-22 KR KR1020247009185A patent/KR20240049332A/ko unknown
-
2024
- 2024-02-12 CO CONC2024/0001464A patent/CO2024001464A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20240049332A (ko) | 2024-04-16 |
AU2022332276A1 (en) | 2024-04-04 |
CA3229323A1 (en) | 2023-03-02 |
WO2023028456A1 (en) | 2023-03-02 |
US20230084036A1 (en) | 2023-03-16 |
AR126846A1 (es) | 2023-11-22 |
TW202323274A (zh) | 2023-06-16 |
CN117836319A (zh) | 2024-04-05 |
IL310997A (en) | 2024-04-01 |
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