CA3207601A1 - Rna adeno-associated virus (raav) vector and uses thereof - Google Patents
Rna adeno-associated virus (raav) vector and uses thereof Download PDFInfo
- Publication number
- CA3207601A1 CA3207601A1 CA3207601A CA3207601A CA3207601A1 CA 3207601 A1 CA3207601 A1 CA 3207601A1 CA 3207601 A CA3207601 A CA 3207601A CA 3207601 A CA3207601 A CA 3207601A CA 3207601 A1 CA3207601 A1 CA 3207601A1
- Authority
- CA
- Canada
- Prior art keywords
- sequence
- rna
- aav
- transcribed
- dna virus
- 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
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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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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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
- 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
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2021075874 | 2021-02-07 | ||
| CNPCT/CN2021/075874 | 2021-02-07 | ||
| PCT/CN2022/075366 WO2022166954A1 (en) | 2021-02-07 | 2022-02-07 | Rna adeno-associated virus (raav) vector and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3207601A1 true CA3207601A1 (en) | 2022-08-11 |
Family
ID=74946911
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3207601A Pending CA3207601A1 (en) | 2021-02-07 | 2022-02-07 | Rna adeno-associated virus (raav) vector and uses thereof |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12031148B2 (enExample) |
| EP (1) | EP4288112A4 (enExample) |
| JP (1) | JP2024508696A (enExample) |
| KR (1) | KR20230142776A (enExample) |
| CN (1) | CN115209924A (enExample) |
| AU (1) | AU2022217349A1 (enExample) |
| BR (1) | BR112023015769A2 (enExample) |
| CA (1) | CA3207601A1 (enExample) |
| IL (1) | IL305002A (enExample) |
| WO (1) | WO2022166954A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116218904B (zh) * | 2022-11-03 | 2025-04-15 | 国药中生生物技术研究院有限公司 | 一种可提高工程化细胞外泌体中特定核酸分子载量的方法及其应用 |
| WO2024120528A1 (en) * | 2022-12-08 | 2024-06-13 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Improved system for producing rna-packaged aav particles |
| CN117778431B (zh) * | 2023-12-04 | 2024-10-25 | 广州派真生物技术有限公司 | 一种用于重组腺相关病毒包装的质粒系统及其应用 |
| WO2025124549A1 (en) * | 2023-12-13 | 2025-06-19 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Improved system and method for producing recombinant dna-packaged aav particles |
| CN117701638B (zh) * | 2023-12-18 | 2024-10-18 | 苏州新芽基因生物技术有限公司 | 一种抑制残留aav载体质粒和质粒骨架转录活性的方法 |
| WO2026095803A1 (en) * | 2024-11-04 | 2026-05-07 | Umc Utrecht Holding B.V. | Sirna delivery with biological vectors |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6346415B1 (en) * | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| JP6738728B2 (ja) * | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| EP3957731A1 (en) * | 2015-07-15 | 2022-02-23 | Rutgers, The State University of New Jersey | Nuclease-independent targeted gene editing platform and uses thereof |
| US11352647B2 (en) * | 2016-08-17 | 2022-06-07 | The Broad Institute, Inc. | Crispr enzymes and systems |
| WO2018035387A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| EP3565608A4 (en) * | 2017-01-05 | 2020-12-16 | Rutgers, The State University of New Jersey | TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES |
| JP7215716B2 (ja) * | 2017-01-13 | 2023-01-31 | 学校法人自治医科大学 | 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター |
| CN111328290A (zh) * | 2017-06-26 | 2020-06-23 | 博德研究所 | 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法 |
| SG11201912024RA (en) * | 2017-07-31 | 2020-02-27 | Sigma Aldrich Co Llc | Synthetic guide rna for crispr/cas activator systems |
| CN109055375B (zh) * | 2018-07-26 | 2021-11-12 | 东南大学 | 一种crispr辅助反式增强子激活基因表达的方法及其应用 |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| MX2023001164A (es) * | 2020-07-29 | 2023-09-15 | Univ America Catholic | Diseño de virus artificiales a base de bacteriofagos para la remodelación del genoma humano. |
-
2022
- 2022-02-07 KR KR1020237030365A patent/KR20230142776A/ko active Pending
- 2022-02-07 CA CA3207601A patent/CA3207601A1/en active Pending
- 2022-02-07 IL IL305002A patent/IL305002A/en unknown
- 2022-02-07 BR BR112023015769A patent/BR112023015769A2/pt not_active Application Discontinuation
- 2022-02-07 JP JP2023548288A patent/JP2024508696A/ja active Pending
- 2022-02-07 EP EP22749233.7A patent/EP4288112A4/en active Pending
- 2022-02-07 WO PCT/CN2022/075366 patent/WO2022166954A1/en not_active Ceased
- 2022-02-07 CN CN202280002654.0A patent/CN115209924A/zh active Pending
- 2022-02-07 AU AU2022217349A patent/AU2022217349A1/en active Pending
- 2022-06-09 US US17/836,222 patent/US12031148B2/en active Active
-
2024
- 2024-05-15 US US18/664,495 patent/US20240368629A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL305002A (en) | 2023-10-01 |
| US12031148B2 (en) | 2024-07-09 |
| WO2022166954A1 (en) | 2022-08-11 |
| US20240368629A1 (en) | 2024-11-07 |
| EP4288112A1 (en) | 2023-12-13 |
| JP2024508696A (ja) | 2024-02-28 |
| EP4288112A4 (en) | 2025-08-06 |
| KR20230142776A (ko) | 2023-10-11 |
| BR112023015769A2 (pt) | 2023-12-12 |
| CN115209924A (zh) | 2022-10-18 |
| US20230062529A1 (en) | 2023-03-02 |
| AU2022217349A1 (en) | 2023-08-31 |
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