SG11202107421YA - Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a - Google Patents
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia aInfo
- Publication number
- SG11202107421YA SG11202107421YA SG11202107421YA SG11202107421YA SG11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA
- Authority
- SG
- Singapore
- Prior art keywords
- hemophilia
- gene therapy
- viral vectors
- increased expression
- vectors encoding
- Prior art date
Links
Classifications
-
- 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
-
- 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
- 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
-
- 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
-
- 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
- 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
-
- 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)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
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 |
|---|---|
| SG11202107421YA true SG11202107421YA (en) | 2021-08-30 |
Family
ID=69593774
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202107421YA SG11202107421YA (en) | 2019-01-16 | 2020-01-15 | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US11707536B2 (pt) |
| EP (1) | EP3911672A1 (pt) |
| JP (1) | JP2022517267A (pt) |
| KR (1) | KR20210117291A (pt) |
| CN (1) | CN113614104A (pt) |
| AR (1) | AR117825A1 (pt) |
| AU (1) | AU2020208375A1 (pt) |
| BR (1) | BR112021013874A2 (pt) |
| CA (1) | CA3127065A1 (pt) |
| CL (1) | CL2021001880A1 (pt) |
| CO (1) | CO2021009338A2 (pt) |
| EA (1) | EA202191977A1 (pt) |
| EC (1) | ECSP21060605A (pt) |
| IL (1) | IL284648A (pt) |
| MX (1) | MX2021008550A (pt) |
| PE (1) | PE20220429A1 (pt) |
| SG (1) | SG11202107421YA (pt) |
| TW (1) | TW202039546A (pt) |
| WO (1) | WO2020150375A1 (pt) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11795207B2 (en) | 2021-03-30 | 2023-10-24 | AAVnerGene Inc. | Modified plasma clotting factor VIII and method of use thereof |
| WO2022211791A1 (en) * | 2021-03-30 | 2022-10-06 | AAVnerGene Inc. | Modified plasma clotting factor viii and method of use thereof |
| TW202400796A (zh) * | 2022-03-11 | 2024-01-01 | 美商同源醫藥公司 | 雙向雙重啟動子表現載體及其用途 |
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 |
| AU5772886A (en) | 1985-04-12 | 1986-11-05 | 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 |
| 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 |
| 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 |
| FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
| CA2162497A1 (en) | 1993-06-10 | 1994-12-22 | Sheila Connelly | Adenoviral vectors for treatment of hemophilia |
| 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 |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| 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 |
| EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| 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 |
| EP2037892B1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| JP5448839B2 (ja) | 2006-12-22 | 2014-03-19 | ツェー・エス・エル・ベーリング・ゲー・エム・ベー・ハー | インビボで長い半減期を有する修飾された凝固因子 |
| KR100959454B1 (ko) | 2007-12-10 | 2010-05-25 | 주식회사 동부하이텍 | 반도체 소자 및 그 제조 방법 |
| JP5797551B2 (ja) | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| US20120093840A1 (en) | 2009-04-06 | 2012-04-19 | Novo Nordisk A/S | Targeted delivery of factor viii proteins to platelets |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| DK2591099T3 (da) | 2010-07-09 | 2021-02-15 | Bioverativ Therapeutics Inc | Kimære koagulationsfaktorer |
| US20130017997A1 (en) | 2010-08-19 | 2013-01-17 | Amunix Operating Inc. | Factor VIII Compositions and Methods of Making and Using Same |
| EA028914B1 (ru) | 2011-07-25 | 2018-01-31 | Байоджен Хемофилия Инк. | Исследования для мониторинга нарушений свертываемости крови |
| ES2700583T3 (es) | 2012-01-12 | 2019-02-18 | Bioverativ Therapeutics Inc | Procedimientos para reducir la inmunogenicidad contra el Factor VIII en individuos sometidos a terapia con Factor VIII |
| KR102212098B1 (ko) | 2012-01-12 | 2021-02-03 | 바이오버라티브 테라퓨틱스 인크. | 키메라 인자 viii 폴리펩티드들과 이의 용도 |
| SG11201404885RA (en) | 2012-02-15 | 2014-09-26 | Amunix Operating Inc | Factor viii compositions and methods of making and using same |
| WO2013123503A1 (en) | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| WO2013151666A2 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| WO2014008172A2 (en) | 2012-07-03 | 2014-01-09 | Expression Therapeutics, Llc | High yield suspension cell line, system and method for making same |
| 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 |
| DK3889173T3 (da) | 2013-02-15 | 2023-10-09 | Bioverativ Therapeutics Inc | Optimeret faktor viii-gen |
| CN105209487A (zh) | 2013-03-15 | 2015-12-30 | 拜耳医药保健有限公司 | 重组因子viii制剂 |
| JP6735672B2 (ja) | 2013-09-12 | 2020-08-05 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター |
| WO2016025764A2 (en) | 2014-08-13 | 2016-02-18 | The Children's Hospital Of Philadelphia | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
| EP3253786A4 (en) | 2015-02-06 | 2018-10-17 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| DK3270944T3 (da) | 2015-03-17 | 2020-01-27 | Univ Brussel Vrije | Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX |
| TW202302627A (zh) | 2015-11-13 | 2023-01-16 | 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 |
| CN112912112A (zh) * | 2018-10-26 | 2021-06-04 | Vrije布鲁塞尔大学 | 肝特异性核酸调节元件以及其方法及用途 |
-
2020
- 2020-01-13 TW TW109101061A patent/TW202039546A/zh unknown
- 2020-01-15 JP JP2021541022A patent/JP2022517267A/ja active Pending
- 2020-01-15 CN CN202080014260.8A patent/CN113614104A/zh active Pending
- 2020-01-15 KR KR1020217025460A patent/KR20210117291A/ko unknown
- 2020-01-15 EP EP20705834.8A patent/EP3911672A1/en active Pending
- 2020-01-15 CA CA3127065A patent/CA3127065A1/en active Pending
- 2020-01-15 WO PCT/US2020/013722 patent/WO2020150375A1/en active Application Filing
- 2020-01-15 EA EA202191977A patent/EA202191977A1/ru unknown
- 2020-01-15 AU AU2020208375A patent/AU2020208375A1/en active Pending
- 2020-01-15 US US16/743,850 patent/US11707536B2/en active Active
- 2020-01-15 PE PE2021001192A patent/PE20220429A1/es unknown
- 2020-01-15 SG SG11202107421YA patent/SG11202107421YA/en unknown
- 2020-01-15 MX MX2021008550A patent/MX2021008550A/es unknown
- 2020-01-15 BR BR112021013874-9A patent/BR112021013874A2/pt unknown
- 2020-01-16 AR ARP200100113A patent/AR117825A1/es unknown
-
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
-
2022
- 2022-12-22 US US18/145,784 patent/US20230211017A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20210117291A (ko) | 2021-09-28 |
| CO2021009338A2 (es) | 2021-07-30 |
| AU2020208375A1 (en) | 2021-07-29 |
| IL284648A (en) | 2021-08-31 |
| MX2021008550A (es) | 2021-08-19 |
| US20230211017A1 (en) | 2023-07-06 |
| BR112021013874A2 (pt) | 2021-09-21 |
| AR117825A1 (es) | 2021-08-25 |
| CN113614104A (zh) | 2021-11-05 |
| CA3127065A1 (en) | 2020-07-23 |
| ECSP21060605A (es) | 2022-01-31 |
| TW202039546A (zh) | 2020-11-01 |
| EA202191977A1 (ru) | 2021-12-15 |
| CL2021001880A1 (es) | 2022-01-14 |
| US11707536B2 (en) | 2023-07-25 |
| WO2020150375A1 (en) | 2020-07-23 |
| PE20220429A1 (es) | 2022-03-29 |
| JP2022517267A (ja) | 2022-03-07 |
| US20200289672A1 (en) | 2020-09-17 |
| EP3911672A1 (en) | 2021-11-24 |
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