SG11202109850SA - Non-viral dna vectors and uses thereof for expressing fviii therapeutics - Google Patents
Non-viral dna vectors and uses thereof for expressing fviii therapeuticsInfo
- Publication number
- SG11202109850SA SG11202109850SA SG11202109850SA SG11202109850SA SG 11202109850S A SG11202109850S A SG 11202109850SA SG 11202109850S A SG11202109850S A SG 11202109850SA SG 11202109850S A SG11202109850S A SG 11202109850SA
- Authority
- SG
- Singapore
- Prior art keywords
- cedna vector
- sequence
- vector
- cedna
- fviii
- 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
- 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
- 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
-
- 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
-
- 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
- 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 |
|---|---|
| SG11202109850SA true SG11202109850SA (en) | 2021-10-28 |
Family
ID=72426821
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202109850S SG11202109850SA (en) | 2019-03-13 | 2020-03-13 | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
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 |
|---|---|---|---|---|
| TW202545984A (zh) | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| US20220356490A1 (en) * | 2020-08-23 | 2022-11-10 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
| US20250027107A1 (en) | 2021-10-18 | 2025-01-23 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| US20240335564A1 (en) * | 2021-11-02 | 2024-10-10 | Gritgen Therapeutics Limited | Isolated nucleic acid molecule and use thereof |
| 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 |
|---|---|---|---|---|
| WO2009067548A1 (en) * | 2007-11-19 | 2009-05-28 | The Regents Of The University Of California | Novel assay for inhibitors of egfr |
| JP2012525134A (ja) * | 2009-04-30 | 2012-10-22 | サイトス バイオテクノロジー アーゲー | インフルエンザ赤血球凝集素の組成物とその使用 |
| 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 |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| SI3889173T1 (sl) | 2013-02-15 | 2023-11-30 | Bioverativ Therapeutics Inc. | Optimiran gen dejavnika VIII |
| RS61039B1 (sr) * | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| GB2526339A (en) | 2014-05-21 | 2015-11-25 | Imp Innovations Ltd | Lentiviral vectors |
| KR20180034467A (ko) * | 2015-08-03 | 2018-04-04 | 마이오도파 리미티드 | L-dopa의 전신 합성 및 조절 |
| RU2742352C2 (ru) | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| CA3002524A1 (en) * | 2015-10-28 | 2017-05-04 | 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 |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| KR102219859B1 (ko) * | 2016-06-24 | 2021-02-25 | 재단법인 목암생명과학연구소 | 재조합 단쇄 fviii 및 그 화학 접합물 |
| AU2018312565A1 (en) * | 2017-08-01 | 2020-02-27 | Spark Therapeutics, Inc. | Factor VIII (FVIII) gene therapy methods |
| TW202545984A (zh) * | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
-
2020
- 2020-03-13 WO PCT/US2020/022738 patent/WO2020186207A2/en not_active Ceased
- 2020-03-13 CN CN202080036209.7A patent/CN113874513A/zh active Pending
- 2020-03-13 SG SG11202109850S patent/SG11202109850SA/en unknown
- 2020-03-13 US US17/437,123 patent/US12338275B2/en active Active
- 2020-03-13 KR KR1020217029545A patent/KR20210151785A/ko active Pending
- 2020-03-13 EP EP20769344.1A patent/EP3938523A4/en active Pending
- 2020-03-13 CA CA3133255A patent/CA3133255A1/en active Pending
- 2020-03-13 MA MA055313A patent/MA55313A/fr unknown
- 2020-03-13 MX MX2021011039A patent/MX2021011039A/es unknown
- 2020-03-13 AU AU2020234713A patent/AU2020234713A1/en active Pending
- 2020-03-13 BR BR112021017853A patent/BR112021017853A2/pt unknown
- 2020-03-13 JP JP2021554696A patent/JP7795357B2/ja active Active
-
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 |
|---|---|
| WO2020186207A2 (en) | 2020-09-17 |
| JP2026002894A (ja) | 2026-01-08 |
| US20250282848A1 (en) | 2025-09-11 |
| KR20210151785A (ko) | 2021-12-14 |
| JP2022524434A (ja) | 2022-05-02 |
| JP2025073123A (ja) | 2025-05-12 |
| EP3938523A4 (en) | 2023-06-21 |
| US20220177545A1 (en) | 2022-06-09 |
| JP7795357B2 (ja) | 2026-01-07 |
| IL286284A (en) | 2021-10-31 |
| MA55313A (fr) | 2022-01-19 |
| US12338275B2 (en) | 2025-06-24 |
| EP3938523A2 (en) | 2022-01-19 |
| CA3133255A1 (en) | 2020-09-17 |
| MX2021011039A (es) | 2021-12-15 |
| BR112021017853A2 (pt) | 2021-11-30 |
| CN113874513A (zh) | 2021-12-31 |
| WO2020186207A8 (en) | 2020-11-12 |
| AU2020234713A1 (en) | 2021-11-04 |
| WO2020186207A3 (en) | 2020-12-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| SG11202109850SA (en) | Non-viral dna vectors and uses thereof for expressing fviii therapeutics | |
| US20220119840A1 (en) | Closed-ended dna (cedna) and use in methods of reducing gene or nucleic acid therapy related immune response | |
| US12037617B2 (en) | Methods and compositions for modulating a genome | |
| US20220042035A1 (en) | Non-viral dna vectors and uses thereof for antibody and fusion protein production | |
| US20220175968A1 (en) | Non-active lipid nanoparticles with non-viral, capsid free dna | |
| BR112020009858A2 (pt) | edição de genes com o uso de um dna modificado com extremidades fechadas (cedna) | |
| US12415002B2 (en) | Non-viral DNA vectors and uses thereof for expressing phenylalanine hydroxylase (PAH) therapeutics | |
| BR112021000145A2 (pt) | Composições de fusossoma e usos das mesmas | |
| UA130154C2 (uk) | Спосіб безклітинного одержання днк-вектора з замкненими кінцями | |
| KR20200051011A (ko) | 변형된 폐쇄-종결된 dna(cedna) | |
| JP2024533313A (ja) | Ｈｂｂを調節する組成物及び方法 | |
| US20230138409A1 (en) | Non-viral dna vectors and uses thereof for expressing factor ix therapeutics | |
| US20240181085A1 (en) | Non-viral dna vectors and uses thereof for expressing pfic therapeutics | |
| US20230134550A1 (en) | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics | |
| US20240026374A1 (en) | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) | |
| WO2024192299A2 (en) | Serpina-modulating compositions and methods | |
| US20230383311A1 (en) | Non-viral dna vectors and uses thereof for expressing fviii therapeutics | |
| CN118556123A (zh) | Hbb调节性组合物和方法 | |
| CN118613588A (zh) | Serpina调节组合物和方法 |