MX2021001375A - Parametros clinicos mejorados por la expresion del factor viii. - Google Patents
Parametros clinicos mejorados por la expresion del factor viii.Info
- Publication number
- MX2021001375A MX2021001375A MX2021001375A MX2021001375A MX2021001375A MX 2021001375 A MX2021001375 A MX 2021001375A MX 2021001375 A MX2021001375 A MX 2021001375A MX 2021001375 A MX2021001375 A MX 2021001375A MX 2021001375 A MX2021001375 A MX 2021001375A
- Authority
- MX
- Mexico
- Prior art keywords
- expression
- factor viii
- clinical parameters
- improved clinical
- improved
- Prior art date
Links
Classifications
-
- 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
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- 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
-
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—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
- C12N2750/14071—Demonstrated in vivo effect
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Hematology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714553P | 2018-08-03 | 2018-08-03 | |
| US201962826887P | 2019-03-29 | 2019-03-29 | |
| US201962869445P | 2019-07-01 | 2019-07-01 | |
| PCT/US2019/044946 WO2020028830A1 (en) | 2018-08-03 | 2019-08-02 | Improved clinical parameters by expression of factor viii |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021001375A true MX2021001375A (es) | 2021-04-19 |
Family
ID=69230742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021001375A MX2021001375A (es) | 2018-08-03 | 2019-08-02 | Parametros clinicos mejorados por la expresion del factor viii. |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US12497632B2 (OSRAM) |
| EP (1) | EP3829650A4 (OSRAM) |
| JP (1) | JP7684212B2 (OSRAM) |
| KR (1) | KR20210039428A (OSRAM) |
| CN (1) | CN112533645B (OSRAM) |
| AU (1) | AU2019314513B2 (OSRAM) |
| BR (1) | BR112021001315A2 (OSRAM) |
| CA (1) | CA3105382A1 (OSRAM) |
| IL (1) | IL280402B2 (OSRAM) |
| MX (1) | MX2021001375A (OSRAM) |
| WO (1) | WO2020028830A1 (OSRAM) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2023006471A (es) * | 2020-12-18 | 2023-08-11 | Sangamo Therapeutics Inc | Composiciones farmaceuticas mejoradas que contienen vector viral adenoasociado. |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1998024479A1 (en) | 1996-12-02 | 1998-06-11 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US7238346B2 (en) | 2002-07-08 | 2007-07-03 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | High capacity recombinant adenoviral vector for treatment of hemophilia A |
| ATE484591T1 (de) | 2003-10-21 | 2010-10-15 | Merck Serono Sa | Minimale dna sequenz, die als chromatin-isolator wirkt, und deren verwendung für die protein- expression |
| WO2006043354A1 (ja) | 2004-10-20 | 2006-04-27 | National Institute Of Radiological Sciences | 組込型の低線量放射線誘導性ベクター |
| JP2008543283A (ja) | 2005-06-10 | 2008-12-04 | ジェネンテック・インコーポレーテッド | インスレーターエレメントを含むベクターを使用する抗体発現の改良 |
| JP2011502478A (ja) | 2007-11-01 | 2011-01-27 | ユニバーシティー オブ ロチェスター | 安定性が増大した組換え型第ｖｉｉｉ因子 |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| US9150847B2 (en) | 2011-09-21 | 2015-10-06 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| JP2015501844A (ja) * | 2011-12-16 | 2015-01-19 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物 |
| EP2804623B1 (en) | 2012-01-12 | 2019-08-07 | Bioverativ Therapeutics Inc. | Chimeric factor viii polypeptides and uses thereof |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| JP2015529466A (ja) | 2012-09-14 | 2015-10-08 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター |
| AU2013336601B2 (en) | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| AU2014246731A1 (en) | 2013-03-30 | 2015-11-19 | Usha Biotech Limited | Methods and constructs for expressing biologically active proteins in mammalian cells |
| EP3770169A1 (en) | 2013-09-12 | 2021-01-27 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
| ES2813367T3 (es) | 2013-12-09 | 2021-03-23 | Sangamo Therapeutics Inc | Métodos y composiciones para ingeniería genómica |
| SG11201605242YA (en) | 2014-01-10 | 2016-07-28 | Biogen Ma Inc | Factor viii chimeric proteins and uses thereof |
| WO2016127057A1 (en) | 2015-02-06 | 2016-08-11 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| EP3283126B1 (en) * | 2015-04-16 | 2019-11-06 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| AU2016259710B2 (en) | 2015-05-11 | 2020-08-27 | Basf Se | Herbicide combinations comprising L-glufosinate and indaziflam |
| CN108024544B (zh) | 2015-07-13 | 2022-04-29 | 桑格摩生物治疗股份有限公司 | 用于核酸酶介导的基因组工程化的递送方法及组合物 |
| PE20181338A1 (es) * | 2015-09-24 | 2018-08-21 | Biomarin Pharm Inc | Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden |
| CN115141836B (zh) * | 2015-10-28 | 2024-08-27 | 桑格摩生物治疗股份有限公司 | 肝特异性构建体、因子viii表达盒及其使用方法 |
| CN116479001A (zh) * | 2015-11-13 | 2023-07-25 | 武田药品工业株式会社 | 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 |
| KR20240160648A (ko) * | 2017-08-01 | 2024-11-11 | 스파크 테라퓨틱스, 인코포레이티드 | 인자 viii (fviii) 유전자 요법 방법 |
-
2019
- 2019-08-02 EP EP19843874.9A patent/EP3829650A4/en active Pending
- 2019-08-02 KR KR1020217006266A patent/KR20210039428A/ko active Pending
- 2019-08-02 CN CN201980051349.9A patent/CN112533645B/zh active Active
- 2019-08-02 MX MX2021001375A patent/MX2021001375A/es unknown
- 2019-08-02 US US17/265,750 patent/US12497632B2/en active Active
- 2019-08-02 AU AU2019314513A patent/AU2019314513B2/en active Active
- 2019-08-02 BR BR112021001315-6A patent/BR112021001315A2/pt unknown
- 2019-08-02 JP JP2021504376A patent/JP7684212B2/ja active Active
- 2019-08-02 WO PCT/US2019/044946 patent/WO2020028830A1/en not_active Ceased
- 2019-08-02 IL IL280402A patent/IL280402B2/en unknown
- 2019-08-02 CA CA3105382A patent/CA3105382A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020028830A1 (en) | 2020-02-06 |
| CN112533645B (zh) | 2026-01-23 |
| AU2019314513B2 (en) | 2026-04-30 |
| JP2021533098A (ja) | 2021-12-02 |
| IL280402B1 (en) | 2026-01-01 |
| BR112021001315A2 (pt) | 2021-04-27 |
| CN112533645A (zh) | 2021-03-19 |
| IL280402A (en) | 2021-03-01 |
| US12497632B2 (en) | 2025-12-16 |
| EP3829650A1 (en) | 2021-06-09 |
| US20210171982A1 (en) | 2021-06-10 |
| KR20210039428A (ko) | 2021-04-09 |
| AU2019314513A1 (en) | 2021-01-28 |
| CA3105382A1 (en) | 2020-02-06 |
| IL280402B2 (en) | 2026-05-01 |
| EP3829650A4 (en) | 2022-05-04 |
| JP7684212B2 (ja) | 2025-05-27 |
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