BR112021001315A2 - parâmetros clínicos melhorados através da expressão do fator viii - Google Patents
parâmetros clínicos melhorados através da expressão do fator viii Download PDFInfo
- Publication number
- BR112021001315A2 BR112021001315A2 BR112021001315-6A BR112021001315A BR112021001315A2 BR 112021001315 A2 BR112021001315 A2 BR 112021001315A2 BR 112021001315 A BR112021001315 A BR 112021001315A BR 112021001315 A2 BR112021001315 A2 BR 112021001315A2
- Authority
- BR
- Brazil
- Prior art keywords
- administration
- fviii
- human
- aav
- seq
- 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
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714553P | 2018-08-03 | 2018-08-03 | |
| US62/714,553 | 2018-08-03 | ||
| US201962826887P | 2019-03-29 | 2019-03-29 | |
| US62/826,887 | 2019-03-29 | ||
| US201962869445P | 2019-07-01 | 2019-07-01 | |
| US62/869,445 | 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 |
|---|---|
| BR112021001315A2 true BR112021001315A2 (pt) | 2021-04-27 |
Family
ID=69230742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021001315-6A BR112021001315A2 (pt) | 2018-08-03 | 2019-08-02 | parâmetros clínicos melhorados através da expressão do fator viii |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12497632B2 (https=) |
| EP (1) | EP3829650A4 (https=) |
| JP (1) | JP7684212B2 (https=) |
| KR (1) | KR20210039428A (https=) |
| CN (1) | CN112533645B (https=) |
| BR (1) | BR112021001315A2 (https=) |
| CA (1) | CA3105382A1 (https=) |
| IL (1) | IL280402B1 (https=) |
| MX (1) | MX2021001375A (https=) |
| WO (1) | WO2020028830A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240066146A1 (en) | 2020-12-18 | 2024-02-29 | Sangamo Therapeutics, Inc. | Improved pharmaceutical compositions containing adeno-associated viral vector |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU5588298A (en) | 1996-12-02 | 1998-06-29 | 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 |
| PT1675956E (pt) | 2003-10-21 | 2010-11-04 | Merck Serono Sa | Sequência mínima de adn que actua como um isolador de cromatina e sua utilização na expressão de proteína |
| JPWO2006043354A1 (ja) | 2004-10-20 | 2008-05-22 | 独立行政法人放射線医学総合研究所 | 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 |
| CN101965409A (zh) | 2007-11-01 | 2011-02-02 | 罗切斯特大学 | 具有增加的稳定性的重组因子viii |
| CA2722238C (en) | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| 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 |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3018046A1 (en) * | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| CN104271150A (zh) | 2012-01-12 | 2015-01-07 | 比奥根艾迪克Ma公司 | 嵌合因子viii多肽及其用途 |
| EP3196301B1 (en) | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic 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 |
| EP3044231B1 (en) | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| CN105940013B (zh) | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| AU2015204646B2 (en) | 2014-01-10 | 2020-08-27 | Bioverativ Therapeutics Inc. | Factor VIII chimeric proteins and uses thereof |
| MX388739B (es) | 2015-02-06 | 2025-03-20 | Univ North Carolina Chapel Hill | Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso. |
| EP3283126B1 (en) | 2015-04-16 | 2019-11-06 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| EP3294064A1 (en) | 2015-05-11 | 2018-03-21 | Bayer CropScience AG | Herbicide combinations comprising l-glufosinate and indaziflam |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| TW202446416A (zh) | 2015-09-24 | 2024-12-01 | 美商拜奧馬林製藥公司 | 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 |
| MY189674A (en) | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| EA202190827A1 (ru) * | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| PE20200722A1 (es) * | 2017-08-01 | 2020-07-21 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii) |
-
2019
- 2019-08-02 US US17/265,750 patent/US12497632B2/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 KR KR1020217006266A patent/KR20210039428A/ko active Pending
- 2019-08-02 MX MX2021001375A patent/MX2021001375A/es unknown
- 2019-08-02 WO PCT/US2019/044946 patent/WO2020028830A1/en not_active Ceased
- 2019-08-02 EP EP19843874.9A patent/EP3829650A4/en active Pending
- 2019-08-02 CA CA3105382A patent/CA3105382A1/en active Pending
- 2019-08-02 CN CN201980051349.9A patent/CN112533645B/zh active Active
-
2021
- 2021-01-26 IL IL280402A patent/IL280402B1/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3829650A1 (en) | 2021-06-09 |
| US20210171982A1 (en) | 2021-06-10 |
| EP3829650A4 (en) | 2022-05-04 |
| CN112533645B (zh) | 2026-01-23 |
| AU2019314513A1 (en) | 2021-01-28 |
| WO2020028830A1 (en) | 2020-02-06 |
| JP7684212B2 (ja) | 2025-05-27 |
| KR20210039428A (ko) | 2021-04-09 |
| US12497632B2 (en) | 2025-12-16 |
| JP2021533098A (ja) | 2021-12-02 |
| CA3105382A1 (en) | 2020-02-06 |
| IL280402A (en) | 2021-03-01 |
| CN112533645A (zh) | 2021-03-19 |
| MX2021001375A (es) | 2021-04-19 |
| IL280402B1 (en) | 2026-01-01 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B08E | Application fees: payment of additional fee required [chapter 8.5 patent gazette] |
Free format text: CONFORME PORTARIA 52/2023, O DEPOSITANTE DEVERA COMPLEMENTAR A RETRIBUICAO DA 6A ANUIDADE, DE ACORDO COM TABELA VIGENTE, REFERENTE A GUIA DE RECOLHIMENTO 29409162328429692 UMA VEZ QUE FOI RECOLHIDA DENTRO DO PRAZO EXTRAORDINARIO, MAS COM VALOR DE PRAZO ORDINARIO (VIDE PARECER). |
|
| B08G | Application fees: restoration [chapter 8.7 patent gazette] | ||
| B06W | Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette] |