BR112021017853A2 - Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii - Google Patents
Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviiiInfo
- Publication number
- BR112021017853A2 BR112021017853A2 BR112021017853A BR112021017853A BR112021017853A2 BR 112021017853 A2 BR112021017853 A2 BR 112021017853A2 BR 112021017853 A BR112021017853 A BR 112021017853A BR 112021017853 A BR112021017853 A BR 112021017853A BR 112021017853 A2 BR112021017853 A2 BR 112021017853A2
- Authority
- BR
- Brazil
- Prior art keywords
- cedna vectors
- fviii protein
- methods
- vectors
- viral dna
- Prior art date
Links
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)
-
- 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
-
- 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
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- C12N2820/00—Vectors comprising a special origin of replication system
- C12N2820/60—Vectors comprising a special origin of replication system from viruses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962817904P | 2019-03-13 | 2019-03-13 | |
| US201962856432P | 2019-06-03 | 2019-06-03 | |
| PCT/US2020/022738 WO2020186207A2 (en) | 2019-03-13 | 2020-03-13 | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021017853A2 true BR112021017853A2 (pt) | 2021-11-30 |
Family
ID=72426821
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021017853A BR112021017853A2 (pt) | 2019-03-13 | 2020-03-13 | Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US12338275B2 (https=) |
| EP (1) | EP3938523A4 (https=) |
| JP (3) | JP7795357B2 (https=) |
| KR (1) | KR20210151785A (https=) |
| CN (1) | CN113874513A (https=) |
| AU (1) | AU2020234713A1 (https=) |
| BR (1) | BR112021017853A2 (https=) |
| CA (1) | CA3133255A1 (https=) |
| IL (1) | IL286284A (https=) |
| MA (1) | MA55313A (https=) |
| MX (1) | MX2021011039A (https=) |
| SG (1) | SG11202109850SA (https=) |
| WO (1) | WO2020186207A2 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| US20220356490A1 (en) * | 2020-08-23 | 2022-11-10 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
| WO2023069948A1 (en) | 2021-10-18 | 2023-04-27 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| EP4428237A4 (en) * | 2021-11-02 | 2026-02-25 | Gritgen Therapeutics Ltd | ISOLATED NUCLEIC ACID MOLECULE AND ITS USES |
| JP2023089774A (ja) * | 2021-12-16 | 2023-06-28 | 株式会社三洋物産 | 遊技機 |
| JP2025504404A (ja) * | 2022-01-14 | 2025-02-12 | アンジャリウム バイオサイエンシズ エージー | Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| KR20260011950A (ko) | 2024-07-17 | 2026-01-26 | 이민형 | 수질 시료 채취 드론 |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20100323957A1 (en) * | 2007-11-19 | 2010-12-23 | The Regents Of The University Of California The Office Of The President | Novel assay for inhibitors of egfr |
| NZ596058A (en) * | 2009-04-30 | 2013-08-30 | Cytos Biotechnology Ag | Influenza hemagglutinin compositions and uses thereof |
| EP2500434A1 (en) | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| WO2013093760A2 (en) * | 2011-12-19 | 2013-06-27 | Grifols, S.A. | Compositions, methods, and kits for preparing sialylated recombinant proteins |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| WO2014127215A1 (en) | 2013-02-15 | 2014-08-21 | Biogen Idec Ma Inc. | Optimized factor viii gene |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| GB2526339A (en) * | 2014-05-21 | 2015-11-25 | Imp Innovations Ltd | Lentiviral vectors |
| US20190032079A1 (en) * | 2015-08-03 | 2019-01-31 | Myodopa Limited | Systemic synthesis and regulation of l-dopa |
| 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 |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| MX2018010633A (es) * | 2016-03-03 | 2019-06-13 | Univ Massachusetts | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
| MX2018012537A (es) | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| JP7235511B2 (ja) * | 2016-06-24 | 2023-03-08 | モガム・インスティテュート・フォー・バイオメディカル・リサーチ | 組換え型一本鎖ｆｖｉｉｉおよびその化学コンジュゲート |
| PE20200722A1 (es) | 2017-08-01 | 2020-07-21 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii) |
| NZ762100A (en) * | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
-
2020
- 2020-03-13 SG SG11202109850S patent/SG11202109850SA/en unknown
- 2020-03-13 CN CN202080036209.7A patent/CN113874513A/zh active Pending
- 2020-03-13 US US17/437,123 patent/US12338275B2/en active Active
- 2020-03-13 EP EP20769344.1A patent/EP3938523A4/en active Pending
- 2020-03-13 AU AU2020234713A patent/AU2020234713A1/en active Pending
- 2020-03-13 MA MA055313A patent/MA55313A/fr unknown
- 2020-03-13 MX MX2021011039A patent/MX2021011039A/es unknown
- 2020-03-13 CA CA3133255A patent/CA3133255A1/en active Pending
- 2020-03-13 BR BR112021017853A patent/BR112021017853A2/pt unknown
- 2020-03-13 WO PCT/US2020/022738 patent/WO2020186207A2/en not_active Ceased
- 2020-03-13 JP JP2021554696A patent/JP7795357B2/ja active Active
- 2020-03-13 KR KR1020217029545A patent/KR20210151785A/ko active Pending
-
2021
- 2021-09-12 IL IL286284A patent/IL286284A/en unknown
-
2025
- 2025-02-03 JP JP2025016352A patent/JP2025073123A/ja active Pending
- 2025-05-21 US US19/214,171 patent/US20250282848A1/en active Pending
- 2025-10-14 JP JP2025172911A patent/JP2026002894A/ja not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| KR20210151785A (ko) | 2021-12-14 |
| WO2020186207A3 (en) | 2020-12-17 |
| JP2026002894A (ja) | 2026-01-08 |
| JP2022524434A (ja) | 2022-05-02 |
| SG11202109850SA (en) | 2021-10-28 |
| JP2025073123A (ja) | 2025-05-12 |
| MA55313A (fr) | 2022-01-19 |
| EP3938523A4 (en) | 2023-06-21 |
| WO2020186207A8 (en) | 2020-11-12 |
| US20250282848A1 (en) | 2025-09-11 |
| US12338275B2 (en) | 2025-06-24 |
| EP3938523A2 (en) | 2022-01-19 |
| US20220177545A1 (en) | 2022-06-09 |
| CA3133255A1 (en) | 2020-09-17 |
| IL286284A (en) | 2021-10-31 |
| AU2020234713A1 (en) | 2021-11-04 |
| WO2020186207A2 (en) | 2020-09-17 |
| CN113874513A (zh) | 2021-12-31 |
| JP7795357B2 (ja) | 2026-01-07 |
| MX2021011039A (es) | 2021-12-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112021017853A2 (pt) | Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii | |
| BR112021017852A2 (pt) | Vetores de dna não virais e usos dos mesmos para expressar terapêuticos de fenilalanina hidroxilase (pah) | |
| JP7235702B2 (ja) | 核酸生成及び送達のための方法及び製品 | |
| Chang et al. | Inducible HGF-secreting human umbilical cord blood-derived MSCs produced via TALEN-mediated genome editing promoted angiogenesis | |
| BR112021024786A2 (pt) | Construção de rna, sequência de ácido nucleico, cassete de expressão, vetor recombinante, composição farmacêutica, método para preparar a construção de rna, vacina, método para modificar uma célula ex vivo ou in vitro e célula modificada | |
| Yang et al. | Genetic engineering of human stem cells for enhanced angiogenesis using biodegradable polymeric nanoparticles | |
| CL2019002280A1 (es) | Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235) | |
| BR112021005769A2 (pt) | proteínas de ligação a dll3 e métodos de uso | |
| PH12020551039A1 (en) | Non-viral dna vectors and uses thereof for antibody and fusion protein production | |
| CO2018005215A2 (es) | Construcciones específicas del hígado para expresión del factor viii | |
| MX2022015080A (es) | Variantes de hsd17b13 y usos de las mismas. | |
| JOP20200050A1 (ar) | عوامل RNAi وتركيبات لتثبيط التعبير عن شبيه آنجيوبويتين 3 (ANGPTL3)، وطرق استخدام | |
| CR20200196A (es) | Proteínas trispecìficas y mètodos de uso | |
| AU2018240515A1 (en) | Nucleic acids encoding CRISPR-associated proteins and uses thereof | |
| BR112014018210A2 (pt) | moléculas de ácido nucleico artificiais para expressão de proteína ou peptídeo melhorada | |
| BR112016002753A2 (pt) | anticorpos contra o inibidor-1 de ativador de plasminogênio (pai-1) e usos dos mesmos | |
| BR112014019431A8 (pt) | Método compartimentalizado de distribuição de ácido nucleico e composições e usos desses | |
| BRPI0510475B8 (pt) | adenovírus quimérico recombinante, uso do mesmo no tratamento de câncer e métodos de inibição de crescimento de uma célula de câncer, de fornecimento de uma proteína terapêutica a uma célula e para o isolamento do adenovírus | |
| MX390848B (es) | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. | |
| MX2022011805A (es) | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. | |
| BR112022004047A2 (pt) | Proteínas de ligação multiespecíficas para o tratamento de câncer | |
| BR112012006501A8 (pt) | Polipeptídios e usos dos mesmos | |
| MX2021009696A (es) | Vectores de genoterapia para el tratamiento de la enfermedad de danon. | |
| BR112014024282A2 (pt) | anticorpos ligantes ao receptor b1 da bradicinina | |
| MX2022011806A (es) | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos contra gaucher. |