CA3088984A1 - Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors - Google Patents
Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors Download PDFInfo
- Publication number
- CA3088984A1 CA3088984A1 CA3088984A CA3088984A CA3088984A1 CA 3088984 A1 CA3088984 A1 CA 3088984A1 CA 3088984 A CA3088984 A CA 3088984A CA 3088984 A CA3088984 A CA 3088984A CA 3088984 A1 CA3088984 A1 CA 3088984A1
- Authority
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- Prior art keywords
- itr
- sequence
- sense
- expression cassette
- stranded
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- 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
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14041—Use of virus, viral particle or viral elements as a vector
- C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/103—Plasmid DNA for invertebrates
- C12N2800/105—Plasmid DNA for invertebrates for insects
-
- 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
-
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Mycology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862619392P | 2018-01-19 | 2018-01-19 | |
| US62/619,392 | 2018-01-19 | ||
| PCT/US2019/014122 WO2019143885A1 (en) | 2018-01-19 | 2019-01-18 | Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3088984A1 true CA3088984A1 (en) | 2019-07-25 |
Family
ID=67301565
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3088984A Pending CA3088984A1 (en) | 2018-01-19 | 2019-01-18 | Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12442015B2 (https=) |
| EP (1) | EP3740571A4 (https=) |
| JP (3) | JP2021511047A (https=) |
| KR (2) | KR20250159738A (https=) |
| CN (1) | CN111868242A (https=) |
| AU (1) | AU2019210034B2 (https=) |
| BR (1) | BR112020013319A2 (https=) |
| CA (1) | CA3088984A1 (https=) |
| IL (1) | IL275878B2 (https=) |
| MA (1) | MA51626A (https=) |
| MX (1) | MX2020005790A (https=) |
| MY (1) | MY208589A (https=) |
| PH (1) | PH12020550878A1 (https=) |
| SG (1) | SG11202005271TA (https=) |
| UA (1) | UA130154C2 (https=) |
| WO (1) | WO2019143885A1 (https=) |
Families Citing this family (30)
| 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 |
| KR20200120649A (ko) * | 2018-02-14 | 2020-10-21 | 제너레이션 바이오 컴퍼니 | 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도 |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| KR20210090619A (ko) * | 2018-11-09 | 2021-07-20 | 제너레이션 바이오 컴퍼니 | 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA) |
| GB201905651D0 (en) | 2019-04-24 | 2019-06-05 | Lightbio Ltd | Nucleic acid constructs and methods for their manufacture |
| CA3147414A1 (en) | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral dna vectors |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| US20230138409A1 (en) * | 2020-03-24 | 2023-05-04 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing factor ix therapeutics |
| 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 |
| US20220243201A1 (en) * | 2020-08-23 | 2022-08-04 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
| EP4214309A4 (en) * | 2020-09-16 | 2025-07-16 | Generation Bio Co | CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) |
| GB202014751D0 (en) | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
| GB202014772D0 (en) | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Nucleic acid construct |
| US20240100132A1 (en) * | 2021-01-25 | 2024-03-28 | Prevail Therapeutics, Inc. | Modulation of aav-based gene expression |
| CN112980862A (zh) * | 2021-02-25 | 2021-06-18 | 通用生物系统（安徽）有限公司 | 一种高纯度微环dna的制备方法 |
| JP2024517427A (ja) | 2021-04-20 | 2024-04-22 | アンジャリウム バイオサイエンシズ エージー | アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法 |
| US20240216535A1 (en) * | 2021-04-27 | 2024-07-04 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| AU2022264509A1 (en) * | 2021-04-27 | 2023-12-14 | Generation Bio Co. | Non-viral dna vectors expressing therapeutic antibodies and uses thereof |
| KR20240005903A (ko) * | 2021-05-07 | 2024-01-12 | 제너레이션 바이오 컴퍼니 | 백신 전달을 위한 비바이러스성 dna 벡터 |
| CA3218126A1 (en) * | 2021-05-07 | 2022-11-10 | Matthew MANGANIELLO | Lyophilized non-viral dna vector compositions and uses thereof |
| AU2022422134A1 (en) * | 2021-12-23 | 2024-07-04 | Generation Bio Co. | Scalable and high-purity cell-free synthesis of closed-ended dna vectors |
| 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 |
| GB202204112D0 (en) | 2022-03-23 | 2022-05-04 | Lightbio Ltd | Linear construct |
| EP4293101A1 (en) | 2022-06-14 | 2023-12-20 | Asklepios Biopharmaceutical, Inc. | Reactor with temperature control and method of using the same |
| CA3262218A1 (en) | 2022-07-19 | 2024-01-25 | Rampart Bioscience, Inc. | CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS |
| US20260055426A1 (en) | 2022-08-19 | 2026-02-26 | Generation Bio Co. | CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF |
| KR20250129818A (ko) * | 2022-12-01 | 2025-08-29 | 제너레이션 바이오 컴퍼니 | 합성 단일-가닥 dna 분자 및 이를 생산하고 사용하는 방법 |
| EP4665865A1 (en) | 2023-02-17 | 2025-12-24 | Anjarium Biosciences AG | Methods of making dna molecules and compositions and uses thereof |
| US11993783B1 (en) * | 2023-03-27 | 2024-05-28 | Genecraft Inc. | Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof |
| WO2026085495A1 (en) | 2024-10-18 | 2026-04-23 | Generation Bio Co. | Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US6346415B1 (en) * | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| CA2467647A1 (en) | 2001-11-30 | 2003-06-05 | Innogenetics N.V. | Therapeutic use of antibodies and fragments thereof binding primate ifn-gamma |
| US8071332B2 (en) | 2007-08-10 | 2011-12-06 | Toto Ltd. | Recombinant mammal cells, method of producing thereof, and method of producing proteins of interest |
| US8709778B2 (en) * | 2008-10-28 | 2014-04-29 | Xavier Danthinne | Method of adenoviral vector synthesis |
| 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 |
| WO2014143932A1 (en) * | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| MX2018010633A (es) * | 2016-03-03 | 2019-06-13 | Univ Massachusetts | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
| JP7026248B2 (ja) | 2017-11-21 | 2022-02-25 | ４ベースバイオ ソシエダー リミターダ | 二本鎖ｄｎａを増幅するための方法およびキット |
| KR20200120649A (ko) * | 2018-02-14 | 2020-10-21 | 제너레이션 바이오 컴퍼니 | 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도 |
| JP2022518504A (ja) * | 2019-01-24 | 2022-03-15 | ジェネレーション バイオ カンパニー | 閉端ｄｎａ（ｃｅｄｎａ）ならびに遺伝子療法または核酸療法に関連する免疫応答を低減させる方法における使用 |
| CA3147414A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral dna vectors |
| CA3146966A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Compositions and production of nicked closed-ended dna vectors |
-
2019
- 2019-01-18 US US16/962,005 patent/US12442015B2/en active Active
- 2019-01-18 BR BR112020013319-1A patent/BR112020013319A2/pt unknown
- 2019-01-18 MX MX2020005790A patent/MX2020005790A/es unknown
- 2019-01-18 SG SG11202005271TA patent/SG11202005271TA/en unknown
- 2019-01-18 KR KR1020257036119A patent/KR20250159738A/ko active Pending
- 2019-01-18 EP EP19741445.1A patent/EP3740571A4/en active Pending
- 2019-01-18 MY MYPI2020003241A patent/MY208589A/en unknown
- 2019-01-18 UA UAA202005240A patent/UA130154C2/uk unknown
- 2019-01-18 KR KR1020207023526A patent/KR102879598B1/ko active Active
- 2019-01-18 CA CA3088984A patent/CA3088984A1/en active Pending
- 2019-01-18 MA MA051626A patent/MA51626A/fr unknown
- 2019-01-18 IL IL275878A patent/IL275878B2/en unknown
- 2019-01-18 AU AU2019210034A patent/AU2019210034B2/en active Active
- 2019-01-18 WO PCT/US2019/014122 patent/WO2019143885A1/en not_active Ceased
- 2019-01-18 JP JP2020539779A patent/JP2021511047A/ja active Pending
- 2019-01-18 CN CN201980019414.XA patent/CN111868242A/zh active Pending
-
2020
- 2020-06-11 PH PH12020550878A patent/PH12020550878A1/en unknown
-
2023
- 2023-07-14 JP JP2023116157A patent/JP2023126487A/ja not_active Withdrawn
-
2025
- 2025-05-21 JP JP2025084944A patent/JP2025113389A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| UA130154C2 (uk) | 2025-12-03 |
| MY208589A (en) | 2025-05-19 |
| EP3740571A4 (en) | 2021-12-08 |
| PH12020550878A1 (en) | 2021-04-05 |
| KR102879598B1 (ko) | 2025-11-03 |
| RU2020127017A (ru) | 2022-02-21 |
| MA51626A (fr) | 2020-11-25 |
| JP2025113389A (ja) | 2025-08-01 |
| BR112020013319A2 (pt) | 2020-12-01 |
| US20210071197A1 (en) | 2021-03-11 |
| IL275878B2 (en) | 2026-03-01 |
| IL275878B1 (en) | 2025-11-01 |
| WO2019143885A1 (en) | 2019-07-25 |
| KR20200111726A (ko) | 2020-09-29 |
| MX2020005790A (es) | 2020-10-28 |
| EP3740571A1 (en) | 2020-11-25 |
| JP2023126487A (ja) | 2023-09-07 |
| CN111868242A (zh) | 2020-10-30 |
| JP2021511047A (ja) | 2021-05-06 |
| IL275878A (en) | 2020-08-31 |
| KR20250159738A (ko) | 2025-11-11 |
| AU2019210034A1 (en) | 2020-07-09 |
| US12442015B2 (en) | 2025-10-14 |
| AU2019210034B2 (en) | 2025-03-27 |
| SG11202005271TA (en) | 2020-07-29 |
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