CO2020007046A2 - Plataformas de entrega crispr objetivo - Google Patents
Plataformas de entrega crispr objetivoInfo
- Publication number
- CO2020007046A2 CO2020007046A2 CONC2020/0007046A CO2020007046A CO2020007046A2 CO 2020007046 A2 CO2020007046 A2 CO 2020007046A2 CO 2020007046 A CO2020007046 A CO 2020007046A CO 2020007046 A2 CO2020007046 A2 CO 2020007046A2
- Authority
- CO
- Colombia
- Prior art keywords
- delivery platforms
- target crispr
- crispr delivery
- nme1cas9
- orthologs
- 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
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
-
- 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
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
- C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Manufacturing & Machinery (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762584310P | 2017-11-10 | 2017-11-10 | |
| US201762596375P | 2017-12-08 | 2017-12-08 | |
| US201862667084P | 2018-05-04 | 2018-05-04 | |
| PCT/US2018/060126 WO2019094791A2 (en) | 2017-11-10 | 2018-11-09 | Targeted crispr delivery platforms |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2020007046A2 true CO2020007046A2 (es) | 2020-08-31 |
Family
ID=66438139
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2020/0007046A CO2020007046A2 (es) | 2017-11-10 | 2020-06-10 | Plataformas de entrega crispr objetivo |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US20220389447A9 (enExample) |
| EP (1) | EP3707254A4 (enExample) |
| JP (2) | JP2021502097A (enExample) |
| KR (2) | KR102922730B1 (enExample) |
| CN (1) | CN111868240A (enExample) |
| AU (2) | AU2018364993B2 (enExample) |
| BR (1) | BR112020009268A2 (enExample) |
| CA (1) | CA3082370A1 (enExample) |
| CO (1) | CO2020007046A2 (enExample) |
| IL (2) | IL317505A (enExample) |
| MX (1) | MX2020004777A (enExample) |
| SG (1) | SG11202005103RA (enExample) |
| WO (1) | WO2019094791A2 (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7250031B2 (ja) * | 2018-09-25 | 2023-03-31 | 公益財団法人微生物化学研究会 | 新規ウイルスベクターおよびその製造方法と使用方法 |
| EP3921638A4 (en) | 2019-02-05 | 2022-08-24 | AMMR Joint Venture | EXPANDED CHOICE OF EFFICIENT TARGETED GENE MANIPULATORS |
| US20220298494A1 (en) * | 2019-02-14 | 2022-09-22 | Metagenomi Ip Technologies, Llc | Enzymes with ruvc domains |
| CN110257406B (zh) * | 2019-06-21 | 2022-07-22 | 安徽省农业科学院水稻研究所 | 密码子植物化改造的Plant Nme2Cas9基因及其应用 |
| US12297426B2 (en) | 2019-10-01 | 2025-05-13 | The Broad Institute, Inc. | DNA damage response signature guided rational design of CRISPR-based systems and therapies |
| WO2021122944A1 (en) | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| CN113652411B (zh) * | 2021-07-30 | 2025-01-10 | 复旦大学 | Cas9蛋白、含有Cas9蛋白的基因编辑系统及应用 |
| AU2022335499A1 (en) | 2021-08-27 | 2024-02-22 | Metagenomi Therapeutics, Inc. | Enzymes with ruvc domains |
| WO2023064813A2 (en) * | 2021-10-13 | 2023-04-20 | University Of Massachusetts | Modified guide rnas for neisseria meningitidis cas9 |
| CA3237303A1 (en) | 2021-11-03 | 2023-05-11 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
| IL312507A (en) * | 2021-11-03 | 2024-07-01 | Intellia Therapeutics Inc | Modified guide rnas for gene editing |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| KR20250124819A (ko) | 2022-12-21 | 2025-08-20 | 인텔리아 테라퓨틱스, 인크. | 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법 |
| TW202436622A (zh) | 2023-03-06 | 2024-09-16 | 美商英特利亞醫療公司 | 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法 |
| WO2025038646A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Cd70 car-t compositions and methods for cell-based therapy |
| WO2025038642A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying cd70 |
| WO2025038637A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2) |
| IL326191A (en) | 2023-08-14 | 2026-03-01 | Intellia Therapeutics Inc | Compositions and methods for genetic modification of transforming growth factor beta receptor type 2 (tgfβr2) |
| WO2025255308A1 (en) | 2024-06-07 | 2025-12-11 | Intellia Therapeutics, Inc. | Cd8 co-receptor chimeric polypeptides in tcr cell therapy |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013266968B2 (en) * | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| RU2716420C2 (ru) * | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| EP3666892A1 (en) * | 2013-07-10 | 2020-06-17 | President and Fellows of Harvard College | Orthogonal cas9 proteins for rna-guided gene regulation and editing |
| CA2930877A1 (en) * | 2013-11-18 | 2015-05-21 | Crispr Therapeutics Ag | Crispr-cas system materials and methods |
| JP2016538001A (ja) * | 2013-11-28 | 2016-12-08 | ホライズン・ジェノミクス・ゲーエムベーハー | 体細胞半数体ヒト細胞株 |
| JP6793547B2 (ja) * | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物 |
| EP3080259B1 (en) * | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| WO2016049258A2 (en) * | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| US10190106B2 (en) * | 2014-12-22 | 2019-01-29 | Univesity Of Massachusetts | Cas9-DNA targeting unit chimeras |
| WO2016179038A1 (en) * | 2015-05-01 | 2016-11-10 | Spark Therapeutics, Inc. | ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE |
| US11732258B2 (en) * | 2016-11-02 | 2023-08-22 | President And Fellows Of Harvard College | Engineered guide RNA sequences for in situ detection and sequencing |
| BR112019028146A2 (pt) * | 2017-07-31 | 2020-07-07 | Sigma-Aldrich Co. Llc | rna guia sintético para sistemas ativadores de crispr/cas |
-
2018
- 2018-11-09 IL IL317505A patent/IL317505A/en unknown
- 2018-11-09 IL IL274526A patent/IL274526B2/en unknown
- 2018-11-09 EP EP18876344.5A patent/EP3707254A4/en active Pending
- 2018-11-09 KR KR1020207016690A patent/KR102922730B1/ko active Active
- 2018-11-09 MX MX2020004777A patent/MX2020004777A/es unknown
- 2018-11-09 SG SG11202005103RA patent/SG11202005103RA/en unknown
- 2018-11-09 KR KR1020267003248A patent/KR20260023096A/ko active Pending
- 2018-11-09 US US16/186,352 patent/US20220389447A9/en not_active Abandoned
- 2018-11-09 AU AU2018364993A patent/AU2018364993B2/en active Active
- 2018-11-09 CA CA3082370A patent/CA3082370A1/en active Pending
- 2018-11-09 BR BR112020009268-1A patent/BR112020009268A2/pt unknown
- 2018-11-09 WO PCT/US2018/060126 patent/WO2019094791A2/en not_active Ceased
- 2018-11-09 CN CN201880082218.2A patent/CN111868240A/zh active Pending
- 2018-11-09 JP JP2020525971A patent/JP2021502097A/ja not_active Withdrawn
-
2020
- 2020-06-10 CO CONC2020/0007046A patent/CO2020007046A2/es unknown
-
2023
- 2023-01-06 AU AU2023200084A patent/AU2023200084B2/en active Active
- 2023-12-28 JP JP2023223638A patent/JP2024019727A/ja active Pending
-
2024
- 2024-03-21 US US18/612,111 patent/US20250223611A1/en active Pending
- 2024-12-13 US US18/980,456 patent/US20250197889A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2018364993B2 (en) | 2022-10-06 |
| KR20200080314A (ko) | 2020-07-06 |
| WO2019094791A2 (en) | 2019-05-16 |
| BR112020009268A2 (pt) | 2020-11-17 |
| CN111868240A (zh) | 2020-10-30 |
| MX2020004777A (es) | 2020-10-08 |
| WO2019094791A3 (en) | 2019-06-20 |
| IL274526A (en) | 2020-06-30 |
| IL274526B2 (en) | 2025-05-01 |
| KR102922730B1 (ko) | 2026-02-03 |
| AU2023200084A1 (en) | 2023-02-09 |
| EP3707254A4 (en) | 2021-08-18 |
| IL317505A (en) | 2025-02-01 |
| JP2021502097A (ja) | 2021-01-28 |
| SG11202005103RA (en) | 2020-06-29 |
| US20220389447A9 (en) | 2022-12-08 |
| US20190338308A1 (en) | 2019-11-07 |
| AU2018364993A1 (en) | 2020-06-11 |
| IL274526B1 (en) | 2025-01-01 |
| CA3082370A1 (en) | 2019-05-16 |
| US20250197889A1 (en) | 2025-06-19 |
| AU2023200084B2 (en) | 2025-10-16 |
| KR20260023096A (ko) | 2026-02-20 |
| US20250223611A1 (en) | 2025-07-10 |
| EP3707254A2 (en) | 2020-09-16 |
| JP2024019727A (ja) | 2024-02-09 |
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